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What changed in Arcellx, Inc.'s 10-K — 2024 vs 2025

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Paragraph-level year-over-year comparison of Arcellx, Inc.'s 2024 and 2025 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2025 report.

+636 added−552 removedSource: 10-K (2026-02-26) vs 10-K (2025-02-27)

Top changes in Arcellx, Inc.'s 2025 10-K

636 paragraphs added · 552 removed · 445 edited across 6 sections

Item 1. Business

Business — how the company describes what it does

189 edited+65 added−67 removed309 unchanged
Biggest changeKey highlights from the preliminary data presented for iMMagine-1 as of the October 31, 2024 data cutoff date are as follows: • For the 86 efficacy evaluable patients with median follow-up of 9.5 months, per IMWG criteria: • 97% (83 of 86) overall response rate (ORR) achieved; 1 • 53 of 86 (62%) patients achieved complete response (CR) or a stringent complete response (sCR); and • 70 of 86 (81%) patients achieved very good partial response (VGPR) or higher. • Of those evaluable for MRD testing (n=58), 54 (93%) were MRD-negative at a minimum of 10 -5 sensitivity. • Median progression free survival (PFS), and overall survival (OS) were not reached, as less than half of all dosed subjects had experienced an event of progression or death. • Using the Kaplan-Meier analysis, which calculates the cumulative survival probability in any given length of time through analysis of subjects who have had an event (death or progression) within specified time intervals: • PFS rates, which reflect the percentage of patients who are alive and have not progressed, at 6 and 12 months were 93% and 79%, respectively; • OS rates, which reflect the percentage of patients who are alive at 6 and 12 months were 97% and 97%, respectively. • For the 98 safety evaluable patients, anito-cel continues to be well-tolerated. • No delayed or non-ICANS neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, have been observed in more than 150 patients dosed with anito-cel. • Cytopenias were the most common Grade ≥3 TEAEs; 53 patients (54%) had Grade ≥3 neutropenia, 20 (20%) had Grade ≥3 thrombocytopenia, and 22 (22%) had Grade ≥3 anemia. • 84 of 98 patients (86%) experienced Grade 1 or lower cytokine release syndrome (CRS), including 17 (17%) with no CRS.
Biggest changeOf the MRD evaluable group with sufficient follow up, 54 of 65 patients (83.1%) were MRD-negative for at least 6 months at a minimum of 10 -5 sensitivity. • Median progression free survival (PFS), and overall survival (OS) were not reached, as less than half of all dosed subjects had experienced an event of progression or death. • Using the Kaplan-Meier analysis, which calculates the cumulative survival probability in any given length of time through analysis of subjects who have had an event (death or progression) within specified time intervals: • PFS rates, which reflect the percentage of patients who are alive and have not progressed, at 6, 12, 18, and 24 months were 93%, 82% and 67%, and 62%, respectively; • OS rates, which reflect the percentage of patients who are alive at 6, 12, 18 and 24 months were 96%, 94%, 88%, and 83%, respectively.
We have summarized our preclinical and clinical programs in the pipeline chart below and indicated where such programs are subject to the Kite Collaboration Agreement, which is described in “Licenses and Collaborations” below.
We have summarized our preclinical and clinical programs in the pipeline chart below and indicated where such programs are subject to the Kite Collaboration Agreement, which is described in “Licenses and Collaborations” below.
The process generally involves the following: • Completion of extensive preclinical studies in accordance with applicable regulations, including studies conducted in accordance with good laboratory practice (GLP) requirements; 36 • Submission to the FDA of an IND, which must become effective before human clinical trials may begin; • Approval by an independent institutional review board (IRB), or ethics committee at each clinical trial site before each trial may be initiated; • Performance of adequate and well-controlled human clinical trials in accordance with applicable IND regulations, GCP requirements and other clinical trial-related regulations to establish the potency, purity and safety of the investigational product for each proposed indication; • Submission to the FDA of a BLA; • A determination by the FDA within 60 days of its receipt of a BLA to accept the filing for review; • Satisfactory completion of a FDA pre-approval inspection of the manufacturing facility or facilities where the biologic will be produced to assess compliance with cGMP, requirements to assure that the facilities, methods and controls are adequate to preserve the biologic’s identity, strength, quality and purity; • Potential FDA audit of the preclinical study and/or clinical trial sites that generated the data in support of the BLA; • FDA review and approval of the BLA, including consideration of the views of any FDA advisory committee, prior to any commercial marketing or sale of the biologic in the United States; and • Compliance with any post-approval requirements, including the potential requirement to implement a REMS, and the potential requirement to conduct post-approval studies.
The process generally involves the following: • Completion of extensive preclinical studies in accordance with applicable regulations, including studies conducted in accordance with good laboratory practice (GLP) requirements; • Submission to the FDA of an IND, which must become effective before human clinical trials may begin; • Approval by an independent institutional review board (IRB), or ethics committee at each clinical trial site before each trial may be initiated; • Performance of adequate and well-controlled human clinical trials in accordance with applicable IND regulations, GCP requirements and other clinical trial-related regulations to establish the potency, purity and safety of the investigational product for each proposed indication; • Submission to the FDA of a BLA; • A determination by the FDA within 60 days of its receipt of a BLA to accept the filing for review; • Satisfactory completion of a FDA pre-approval inspection of the manufacturing facility or facilities where the biologic will be produced to assess compliance with cGMP, requirements to assure that the facilities, methods and controls are adequate to preserve the biologic’s identity, strength, quality and purity; • Potential FDA audit of the preclinical study and/or clinical trial sites that generated the data in support of the BLA; • FDA review and approval of the BLA, including consideration of the views of any FDA advisory committee, prior to any commercial marketing or sale of the biologic in the United States; and • Compliance with any post-approval requirements, including the potential requirement to implement a REMS, and the potential requirement to conduct post-approval studies.
Specifically, they may limit the number of patients that are eligible for treatment, complicate adoption into earlier lines of treatment, preclude the use of these therapies in the non-academic and outpatient settings, and increase costs to patients, payers and providers due to the need for intensive care unit access when they are used. • Narrow Applicability : Currently, CAR-T and other genetically modified cell therapies are utilized in only a few hematological oncology indications.
Specifically, they may limit the number of patients that are eligible for treatment, complicate adoption into earlier lines of treatment, 11 preclude the use of these therapies in the non-academic and outpatient settings, and increase costs to patients, payers and providers due to the need for intensive care unit access when they are used. • Narrow Applicability : Currently, CAR-T and other genetically modified cell therapies are utilized in only a few hematological oncology indications.
We are generating D-Domains against multiple targets which can then be deployed to create a new class of D-Domain powered cell therapies, including ddCAR and ARC-SparX CAR-T therapies, to address hematologic cancers, solid tumors, and indications outside of oncology such as autoimmune diseases. ddCARs are single infusion CAR-Ts enhanced with our D-Domains as the antigen recognition motif.
We are generating D-Domains against multiple targets which can then be deployed to create a new class of D-Domain powered CAR-Ts, including ddCAR and ARC-SparX CAR-T therapies, to address hematologic cancers, solid tumors, and indications outside of oncology such as autoimmune diseases. ddCARs are single infusion CAR-Ts enhanced with our D-Domains as the antigen recognition motif.
Upon completion of the Phase 1 trial, we will leverage the learnings from this trial to advance our AML/MDS programs utilizing ARC-SparX and consider developing additional SparX for rrMM for a broader pipeline in this disease area. Additionally, in December 2023, Kite exercised its option under the Kite Collaboration Agreement to negotiate a license for ACLX-001.
Upon completion of the Phase 1 trial, we will leverage the learnings from this trial to advance our AML/MDS programs utilizing ARC-SparX and 32 consider developing additional SparX for rrMM for a broader pipeline in this disease area. Additionally, in December 2023, Kite exercised its option under the Kite Collaboration Agreement to negotiate a license for ACLX-001.
The IMWG criteria for sCR, CR, VGPR, and PR are summarized below. • stringent Complete Response (sCR) : Complete Response (as defined below) plus normal free light chain (FLC) ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry (kappa to lambda light chain ratio ( k / l ) ≤4:1 or ≥1:2 for k or l patients, respectively, after counting ≥100 plasma cells). 20 • Complete Response (CR) : Negative immunofixation in the serum and urine; and disappearance of any soft tissue plasmacytomas; and • Very Good Partial Response (VGPR) : Serum and urine M protein, detectable by immunofixation but not on electrophoresis; or ≥90% reduction in serum M protein plus urine M protein level • Partial Response (PR) : ≥50% reduction of serum M protein plus reduction in 24-hour urinary M protein by ≥90% or to Overall Response Rate (ORR) includes patients that achieved sCR, CR, VGPR or PR. sCR and CR do not indicate that the patient was cured of the condition, as multiple myeloma is currently considered incurable.
The IMWG criteria for sCR, CR, VGPR, and PR are summarized below. • stringent Complete Response (sCR) : Complete Response (as defined below) plus normal free light chain (FLC) ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry (kappa to lambda light chain ratio ( k / l ) ≤4:1 or ≥1:2 for k or l patients, respectively, after counting ≥100 plasma cells). • Complete Response (CR) : Negative immunofixation in the serum and urine; and disappearance of any soft tissue plasmacytomas; and • Very Good Partial Response (VGPR) : Serum and urine M protein, detectable by immunofixation but not on electrophoresis; or ≥90% reduction in serum M protein plus urine M protein level • Partial Response (PR) : ≥50% reduction of serum M protein plus reduction in 24-hour urinary M protein by ≥90% or to 23 Overall Response Rate (ORR) includes patients that achieved sCR, CR, VGPR or PR. sCR and CR do not indicate that the patient was cured of the condition, as multiple myeloma is currently considered incurable.
The process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state, local and foreign statutes and regulations requires the expenditure of substantial time and financial resources. Failure to comply with the applicable U.S. requirements at any time during the product development process, approval process or post-market may subject an applicant to administrative or judicial sanctions.
The process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state, local and foreign statutes and regulations requires the expenditure of substantial time and financial resources. Failure to comply with the applicable U.S. requirements at any time during the product development process, approval process or post-market may subject an applicant to 39 administrative or judicial sanctions.
Clinical Trials The clinical stage of development involves the administration of the investigational product to healthy volunteers or patients under the supervision of qualified investigators, generally physicians not employed by or under the trial sponsor’s control, in accordance with GCP requirements, which include the requirement that all research subjects provide their informed consent for their participation in any clinical trial.
Clinical Trials The clinical stage of development involves the administration of the investigational product to healthy volunteers or patients under the supervision of qualified investigators, generally physicians not employed by or under the trial sponsor’s control, in accordance with GCP requirements, which include the requirement that all research subjects provide their informed consent for their 40 participation in any clinical trial.
Additionally, appropriate packaging must be selected and tested and stability studies must be conducted to demonstrate that the product candidates do not undergo unacceptable deterioration over their shelf life. Compliance with cGMP and GTP Requirements Before approving a BLA, the FDA typically will inspect the facility or facilities where the product is manufactured.
Additionally, appropriate packaging must be selected and tested and stability studies must be conducted to demonstrate that the product candidates do not undergo unacceptable deterioration over their shelf life. Compliance with cGMP and GTP Requirements 41 Before approving a BLA, the FDA typically will inspect the facility or facilities where the product is manufactured.
While all Medicare drug plans must give at least a standard level of coverage set by Medicare, Part D prescription drug plan sponsors are not required to pay for all covered Part D drugs, and each drug plan can develop its own drug formulary that identifies which drugs it will cover and at what tier or level.
While all Medicare drug plans must give at least a standard level of coverage set by 50 Medicare, Part D prescription drug plan sponsors are not required to pay for all covered Part D drugs, and each drug plan can develop its own drug formulary that identifies which drugs it will cover and at what tier or level.
The Surveillance, Epidemiology, and End Results (SEER) Program database projects that approximately 35,000 new cases of MM in the United States and over 35,000 new cases in six select markets within Europe and Asia. The median age of MM patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years.
The Surveillance, Epidemiology, and End Results (SEER) Program database projects that approximately 35,000 new cases of MM in the United States and over 36,000 new cases in six select markets within Europe and Asia. The median age of MM patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years.
Efficacy data are assessed pursuant to the IMWG criteria on a monthly basis for the first 6 months and then quarterly for up to two years, or upon symptomatic relapse. The IMWG uniform response criteria have been utilized in registration trials of approved myeloma drugs.
Efficacy data are assessed pursuant to the IMWG criteria on a monthly basis for the first 6 months and then quarterly for up to two years, or upon symptomatic relapse. 22 The IMWG uniform response criteria have been utilized in registration trials of approved myeloma drugs.
Patients in the control arm will continue to receive the selected SOC regimen. The primary endpoint is progression-free survival (PFS) per blinded independent review according to the IMWG uniform response criteria for multiple myeloma (MM) with the hypothesis that anito-cel will prolong PFS compared to SOC.
Patients in the control arm will continue to receive the selected SOC regimen. 31 The primary endpoint is progression-free survival (PFS) per blinded independent review according to the IMWG uniform response criteria for multiple myeloma (MM) with the hypothesis that anito-cel will prolong PFS compared to SOC.
The small antigen binding domain may also function to improve the immunological synapse formation and thus CAR-T cell killing. • Hydrophobic Core : The figure below depicts the three-dimensional structure of the D-Domain highlighting the triple alpha helical bundle with the tight hydrophobic core (in red).
The small antigen binding domain may also function to improve the immunological synapse formation and thus CAR-T cell killing. 14 • Hydrophobic Core : The figure below depicts the three-dimensional structure of the D-Domain highlighting the triple alpha helical bundle with the tight hydrophobic core (in red).
Rami Elghandour, our Chairman and Chief Executive Officer, previously served as President and Chief Executive Officer at Nevro where he grew the company from a small private company to a publicly traded commercial organization with nearly $400 million in revenue. Prior to Nevro, Mr.
Rami Elghandour, our Chairman and Chief Executive Officer, previously served as President and Chief Executive Officer at Nevro where he grew the company from a small private company to a publicly traded commercial organization with nearly $400 million in revenue. 9 Prior to Nevro, Mr.
The hydrophobic core results in ultrafast folding kinetics of the D-Domain creating a stable structure when expressed in cells. 11 • Stability : D-Domains are highly stable proteins compared to scFvs which facilitates the high expression of CARs on T-cells and manufacturing of SparX proteins.
The hydrophobic core results in ultrafast folding kinetics of the D-Domain creating a stable structure when expressed in cells. • Stability : D-Domains are highly stable proteins compared to scFvs which facilitates the high expression of CARs on T-cells and manufacturing of SparX proteins.
There are two types of Marketing Authorizations: • The Community MA is issued by the European Commission through the Centralized Procedure, based on the opinion of the Committee for Medicinal Products for Human Use (CHMP), of the European Medicines Agency (EMA), and is valid throughout the entire territory of the EEA.
There are two types of Marketing Authorizations: • The Centralized MA is issued by the European Commission through the Centralized Procedure, based on the opinion of the Committee for Medicinal Products for Human Use (CHMP), of the European Medicines Agency (EMA), and is valid throughout the entire territory of the EEA.
Like some of FDA’s other expedited development programs, RMAT designation does not change the standards for approval but may help expedite the development or approval process. 41 Abbreviated Licensure Pathway of Biological Products as Biosimilars or Interchangeable Biosimilars The Patient Protection and Affordable Care Act (ACA), signed into law in 2010, includes the Biologics Price Competition and Innovation Act of 2009 (BPCIA), which created an abbreviated approval pathway for biological products shown to be highly similar to an FDA-licensed reference biological product.
Like some of FDA’s other expedited development programs, RMAT designation does not change the standards for approval but may help expedite the development or approval process. 44 Abbreviated Licensure Pathway of Biological Products as Biosimilars or Interchangeable Biosimilars The Patient Protection and Affordable Care Act (ACA), signed into law in 2010, includes the Biologics Price Competition and Innovation Act of 2009 (BPCIA), which created an abbreviated approval pathway for biological products shown to be highly similar to an FDA-licensed reference biological product.
In the future, we may apply for restoration of patent term for our currently owned or licensed patents to add patent life beyond its current expiration date, depending on the expected length of the clinical trials and other factors involved in the filing of the relevant BLA. 44 Government Regulation Outside of the United States In addition to regulations in the United States, we are subject to a variety of regulations in other jurisdictions where we seek to commercialize any of our product candidates, including countries in Europe and Asia.
In the future, we may apply for restoration of patent term for our currently owned or licensed patents to add patent life beyond its current expiration date, depending on the expected length of the clinical trials and other factors involved in the filing of the relevant BLA. 47 Government Regulation Outside of the United States In addition to regulations in the United States, we are subject to a variety of regulations in other jurisdictions where we seek to commercialize any of our product candidates, including countries in Europe and Asia.
For example, the FDA has discretion over the kind and amount of scientific evidence—laboratory, preclinical and/or clinical—required to demonstrate biosimilarity to a licensed biological product. 42 The timing of final FDA approval of a biosimilar for commercial distribution depends on a variety of factors, including whether the manufacturer of the branded product is entitled to one or more statutory exclusivity periods, during which time the FDA is prohibited from approving any products that are biosimilar to the branded product.
For example, the FDA has discretion over the kind and amount of scientific evidence—laboratory, preclinical and/or clinical—required to demonstrate biosimilarity to a licensed biological product. 45 The timing of final FDA approval of a biosimilar for commercial distribution depends on a variety of factors, including whether the manufacturer of the branded product is entitled to one or more statutory exclusivity periods, during which time the FDA is prohibited from approving any products that are biosimilar to the branded product.
Kite will be solely responsible for the conduct of development of the Non-Co-Promote Products at its sole cost. In the United States, we and Kite will be jointly responsible for commercialization of the Co-Promote Products. Kite will be responsible, at its sole cost, for commercialization of the Co-Promote Products outside the United States and the Non-Co-Promote Products worldwide.
Kite will be solely responsible for the conduct of development of the Non-Co-Promote Products at its sole cost. In the United States, we and Kite will be jointly responsible for commercialization of the Co-Promote Products. Kite will be responsible, at its sole cost, for commercialization of the Co-Promote Products outside the United States and the 37 Non-Co-Promote Products worldwide.
Thus, new therapies are needed for MDS patients as well. 30 ACLX-002 (CD123): Phase 1 Trial Our first AML/MDS product candidate is ACLX-002, which is an immunotherapeutic combination agent composed of the same ARC-T-cells used in ACLX-001, together with mono-valent SparX proteins that each contain a binding domain directed at CD123.
Thus, new therapies are needed for MDS patients as well. 33 ACLX-002 (CD123): Phase 1 Trial Our first AML/MDS product candidate is ACLX-002, which is an immunotherapeutic combination agent composed of the same ARC-T-cells used in ACLX-001, together with mono-valent SparX proteins that each contain a binding domain directed at CD123.
A subgroup analysis of subjects with high risk clinical features (defined as presence of EMD, BMPC ≥ 60%, High Risk Cytogenetics or ISS Stage III) indicated similar PFS rates at 12, 24, and 30 months of 72%, 60%, and 60%, respectively. 23 Of the 38 patients dosed in our Phase 1 trial of anito-cel, all (100%) were evaluable for safety analysis.
A subgroup analysis of subjects with high risk clinical features (defined as presence of EMD, BMPC ≥ 60%, High Risk Cytogenetics or ISS Stage III) indicated similar PFS rates at 12, 24, and 30 months of 72%, 60%, and 60%, respectively. 26 Of the 38 patients dosed in our Phase 1 trial of anito-cel, all (100%) were evaluable for safety analysis.
We also intend to employ clinical and translational strategies such as combinations with checkpoint inhibitors to boost activity of ddCAR or ARC-T-cells to further overcome some common immunological barriers to successful CAR-T therapy. 31 Additional Indications and Applications of Our Technology We believe our platform technologies lend themselves to a broad array of potential applications, including: Novel Targets .
We also intend to employ clinical and translational strategies such as combinations with checkpoint inhibitors to boost activity of ddCAR or ARC-T-cells to further overcome some common immunological barriers to successful CAR-T therapy. 34 Additional Indications and Applications of Our Technology We believe our platform technologies lend themselves to a broad array of potential applications, including: Novel Targets .
We plan to achieve this goal by maximizing the impact of our proprietary D-Domain binders, which may enable CAR-Ts to have distinct advantages that address these limitations, including achieving promising preliminary clinical data with high ORR and durable responses, potentially differentiated safety profile, opportunity to treat a broader group of patients, and potential manufacturability advantages through our D-Domain technology and the partnership with an experienced CAR-T company (see Kite Collaboration Agreement), as detailed in the section entitled “Cell Therapy Background & Current Limitations” below.
We plan to achieve this goal by maximizing the impact of our proprietary D-Domain binders, which may enable CAR-Ts to have distinct advantages that address these limitations, including achieving promising preliminary clinical data with high ORR and durable responses, potentially differentiated safety profile, opportunity to treat a broader group of patients, and potential manufacturability advantages through our D-Domain technology and the partnership with an experienced CAR-T company (see Kite Collaboration Agreement), as detailed in the section entitled “CAR-T Background & Current Limitations” below.
We believe the low propensity for tonic signaling of D-Domain-based CARs may lower T cell exhaustion. 13 • Engineered D-Domain Scaffolds : The structural features of the D-Domain make it particularly well suited as a scaffold protein that can be modified by inserting selected amino acids to generate diverse libraries of proprietary target-binding domains.
We believe the low propensity for tonic signaling of D-Domain-based CARs may lower T cell exhaustion. 17 • Engineered D-Domain Scaffolds : The structural features of the D-Domain make it particularly well suited as a scaffold protein that can be modified by inserting selected amino acids to generate diverse libraries of proprietary target-binding domains.
Applying all these discovery methods, the engineered D-Domains are incorporated into our genetically modified T-cells in our ddCAR and ARC-SparX platform. 14 ARC-SparX Platform Our ARC-SparX platform is a controllable and adaptable modular therapy that builds on our ddCARs platform by replacing the antigen binding domain of the T cell with a novel synthetic binding domain that recognizes only SparX proteins, which contain the antigen binding domain.
Applying all these discovery methods, the engineered D-Domains are incorporated into our genetically modified T-cells in our ddCAR and ARC-SparX platform. 18 ARC-SparX Platform Our ARC-SparX platform is a controllable and adaptable modular therapy that builds on our ddCARs platform by replacing the antigen binding domain of the T cell with a novel synthetic binding domain that recognizes only SparX proteins, which contain the antigen binding domain.
As shown in the right panel of the figure below, our anito-cel transduced T-cells demonstrated superior transduction efficiency when compared to scFv transduced T-cells derived from multiple normal human donors. 12 • High Cell Surface Expression : Coincident with higher transduction rates, the expression of the CAR on the surface of the T cell is higher with CARs employing a D-Domain compared with an scFv.
As shown in the right panel of the figure below, our anito-cel transduced T-cells demonstrated superior transduction efficiency when compared to scFv transduced T-cells derived from multiple normal human donors. 16 • High Cell Surface Expression : Coincident with higher transduction rates, the expression of the CAR on the surface of the T cell is higher with CARs employing a D-Domain compared with an scFv.
Except for such partnered programs, we have worldwide rights to all our programs. 3 * Kite exercised its option to negotiate a license for ACLX-001 and retains one remaining option for a select ARC-SparX program in multiple myeloma and lymphoma Outside our collaboration with Kite, we are also advancing anito-cel into select autoimmune disorders.
Except for such partnered programs, we have worldwide rights to all our programs. * Kite exercised its option to negotiate a license for ACLX-001 and retains one remaining option for a select ARC-SparX program in multiple myeloma and lymphoma 5 Outside our collaboration with Kite, we are also advancing anito-cel into select autoimmune disorders.
The observed AEs are consistent with those of other autologous CAR-T-cells in clinical trials and in commercial use. 24 anito-cel: Phase 2 Pivotal Trial in rrMM (iMMagine-1) Our Phase 2 pivotal trial of anito-cel in rrMM, the iMMagine-1 trial is a single-arm, open-label, evaluation of the efficacy of anito-cel, as measured by the primary endpoint of ORR.
The observed AEs are consistent with those of other autologous CAR-T-cells in clinical trials and in commercial use. 27 anito-cel: Phase 2 Pivotal Trial in rrMM (iMMagine-1) Our Phase 2 pivotal trial of anito-cel in rrMM, the iMMagine-1 trial is a single-arm, open-label, evaluation of the efficacy of anito-cel, as measured by the primary endpoint of ORR.
Further, the Company’s references to the URLs for these websites are intended to be inactive textual references only. We have used, and intend to continue to use, our investor relations website, press releases, public conference calls, and webcasts to disclose material non-public information and to comply with our disclosure obligations under Regulation FD. 49
Further, the Company’s references to the URLs for these websites are intended to be inactive textual references only. We have used, and intend to continue to use, our investor relations website, press releases, public conference calls, and webcasts to disclose material non-public information and to comply with our disclosure obligations under Regulation FD. 52
Genetically modified cell therapy involves isolating immune cells, modifying them outside of the patient’s body and then reintroducing them into the patient to destroy diseased cells. Such cell therapies have largely focused on using the patient’s own T-cells (autologous approach) to express engineered antigen receptor complexes, such as TCRs or CARs.
Genetically modified CAR-T involves isolating immune cells, modifying them outside of the patient’s body and then reintroducing them into the patient to destroy diseased cells. Such cell therapies have largely focused on using the patient’s own T-cells (autologous approach) to express engineered antigen receptor complexes, such as TCRs or CARs.
Our ARC-T-cells are dosable, controllable, universal CAR-Ts designed to activate only when combined with a SparX protein that is bound to an antigen on a cell. 9 ddCAR Platform We use our ddCAR platform to generate single infusion therapies where our D-Domain binder replaces the scFvs.
Our ARC-T-cells are dosable, controllable, universal CAR-Ts designed to activate only when combined with a SparX protein that is bound to an antigen on a cell. 13 ddCAR Platform We use our ddCAR platform to generate single infusion therapies where our D-Domain binder replaces the scFvs.
Cell Therapy Background & Current Limitations Background T-cells are a key component of the immune system that can target diseased cells for elimination through the recognition of cell surface antigens. A growing understanding of the immune system over the years and advances in cell, gene and protein engineering have led to approved genetically modified cell therapy products.
CAR-T Background & Current Limitations Background T-cells are a key component of the immune system that can target diseased cells for elimination through the recognition of cell surface antigens. A growing understanding of the immune system over the years and advances in cell, gene and protein engineering have led to approved genetically modified CAR-T products.
Further, we believe the Kite Collaboration Agreement and the associated economic terms substantially limits our need for additional capital to build out commercial manufacturing infrastructure. The foundation of our proprietary platform is our D-Domain technology, that has generated promising initial clinical data.
Further, we believe the Kite Collaboration Agreement and the associated economic terms substantially limit our need for additional capital to build out commercial manufacturing infrastructure. The foundation of our proprietary platform is our D-Domain technology, that has generated promising initial clinical data.
We believe anito-cel's mechanism in targeting plasma cells through BCMA may be a relevant mechanism to address the underlying pathogenesis of disease. We estimate that gMG affects over 70,000 people in the United States and there is no known cure.
We believe anito-cel's mechanism in targeting plasma cells through BCMA may be a relevant mechanism to address the underlying pathogenesis of disease. We estimate that gMG affects over 100,000 people in the United States and there is no known cure.
The Centralized Procedure is optional for products containing a new active substance not yet authorized in the EEA, or for products that constitute a significant therapeutic, scientific or technical innovation or for products that are in the interest of public health in the European Union. 45 • National MAs, which are issued by the competent authorities of the Member States of the EEA and only cover their respective territory, are available for products not falling within the mandatory scope of the Centralized Procedure.
The Centralized Procedure is optional for products containing a new active substance, or for products that constitute a significant therapeutic, scientific or technical innovation or for products that are in the interest of public health in the European Union. • National MAs, which are issued by the competent authorities of the Member States of the EEA and only cover their respective territory, are available for products not falling within the mandatory scope of the Centralized Procedure.
Our binding domains have many positive attributes over scFv binding domains that we believe could allow them to be used as an scFv alternative in non-cell therapy applications and serve as the foundation to creating a new class of therapeutic antibody alternatives. Manufacturing and Delivery Our manufacturing process is consistent across anito-cel cells and ARC-T-cells.
Our binding domains have many positive attributes over scFv binding domains that we believe could allow them to be used as an scFv alternative in non-CAR-T applications and serve as the foundation to creating a new class of therapeutic antibody alternatives. Manufacturing and Delivery Our manufacturing process is consistent across anito-cel cells and ARC-T-cells.
As of December 31, 2024, the two CAR-T therapies targeting BCMA that have been approved by the FDA are Abecma and Carvykti, developed and marketed by 2seventy bio/Bristol Myers Squibb and Legend/Johnson & Johnson, respectively.
As of December 31, 2025, the two CAR-T therapies targeting BCMA that have been approved by the FDA are Abecma and Carvykti, developed and marketed by 2seventy bio/Bristol Myers Squibb and Legend/Johnson & Johnson, respectively.
In the safety and efficacy analysis, 38 patients were evaluable, 32 in the DL1 and 6 in DL2. Median administered dose at DL1, 115 million cells (range, 112-120 million cells) 21 Patient and Disease Characteristics are demonstrated in the table below.
In the safety and efficacy analysis, 38 patients were evaluable, 32 in the DL1 and 6 in DL2. Median administered dose at DL1, 115 million cells (range, 112-120 million cells) 24 Patient and Disease Characteristics are demonstrated in the table below.
The FDA and other agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to significant liability. 43 Other U.S.
The FDA and other agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to significant liability. 46 Other U.S.
Except for such partnered programs, we have worldwide rights to all of our programs: * Kite exercised its option to negotiate a license for ACLX-001 and retains an option for additional select ARC-SparX programs in multiple myeloma and lymphoma Our Multiple Myeloma Program Our MM program is led by our anito-cel product candidate, which is an autologous cell therapy comprised of D-Domain powered T-cells that have been genetically modified to recognize and kill specific cells expressing BCMA, a target antigen for multiple myeloma.
Except for such partnered programs, we have worldwide rights to all of our programs: * Kite exercised its option to negotiate a license for ACLX-001 and retains an option for additional select ARC-SparX programs in multiple myeloma and lymphoma 20 Our Multiple Myeloma Program Our MM program is led by our anito-cel product candidate, which is an autologous CAR-T comprised of D-Domain powered T-cells that have been genetically modified to recognize and kill specific cells expressing BCMA, a target antigen for multiple myeloma.
Median OS was Not Reached. 22 A Kaplan-Meier analysis of all subjects demonstrated a PFS rate at 6, 12, 18, 24, and 30 months of 92%, 76%, 65%, 57%, and 50%, respectively.
Median OS was Not Reached. 25 A Kaplan-Meier analysis of all subjects demonstrated a PFS rate at 6, 12, 18, 24, and 30 months of 92%, 76%, 65%, 57%, and 50%, respectively.
As of December 31, 2024, our patent portfolio includes four patent families directed to the proprietary D-Domain binding domain technology. • The first patent family broadly covers libraries of our proprietary D-Domain binding domains, compositions comprising our proprietary D-Domain binding domains and methods of using our proprietary D-Domain binding domains.
As of December 31, 2025, our patent portfolio includes four patent families directed to the proprietary D-Domain binding domain technology. • The first patent family broadly covers libraries of our proprietary D-Domain binding domains, compositions comprising our proprietary D-Domain binding domains and methods of using our proprietary D-Domain binding domains.
We believe we can address these limitations by engineering a new class of D-Domain powered cell therapies, including classical single infusion CAR-Ts called “ddCARs” and dosable and controllable universal CAR-Ts called “ARC-SparX”, to address hematologic cancers, solid tumors, and indications outside of oncology, such as autoimmune diseases.
We believe we can address these limitations by engineering a new class of D-Domain powered immunotherapies, including classical single infusion CAR-Ts called “ddCARs” and dosable and controllable universal CAR-Ts called “ARC-SparX”, to address hematologic cancers, solid tumors, and indications outside of oncology, such as autoimmune diseases.
Certain countries outside of the United States have a similar approval process that requires the submission of a clinical trial application, or CTA, much like the IND prior to the commencement of human clinical trials.
Certain countries outside of the United States have a similar approval process that requires the submission of a clinical trial application (CTA), much like the IND prior to the commencement of human clinical trials in the United States.
Although cell therapies have shown benefits to date, cell therapies have primarily been constrained to existing biologic structures, which has limited their impact and opportunity. Our novel synthetic binding scaffold, the D-Domain, is designed to overcome the limitations of traditional Chimeric Antigen Receptor T-cells (CAR-Ts).
Although Chimeric Antigen Receptor T-cells (CAR-Ts) have shown benefits to date, they have primarily been constrained to existing biologic structures, which has limited their impact and opportunity. Our novel synthetic binding scaffold, the D-Domain, is designed to overcome the limitations of traditional CAR-Ts.
For example, across the clinical trials of Abecma and Carvykti, several poor prognostic factors have been identified including the presence of extra-medullary disease (EMD) and more broadly plasmacytomas, ISS stage 3, high tumor burden, and high-risk cytogenetics. In clinical trials in rrMM, these patients demonstrated shorter PFS rates.
Additionally, across the clinical trials of Abecma and Carvykti, several poor prognostic factors have been identified including the presence of extra-medullary disease (EMD) and more broadly plasmacytomas, ISS stage 3, high tumor burden, and high-risk cytogenetics. In clinical trials in rrMM, these patients demonstrated shorter PFS rates.
Mergers and acquisitions in the pharmaceutical, biotechnology, gene therapy and cell therapy industries may result in even more resources being concentrated among a smaller number of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies.
Mergers and acquisitions in the pharmaceutical, biotechnology, gene therapy and CAR-T industries may result in even more resources being concentrated among a smaller number of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies.
Where a product has already been authorized for marketing in a Member State of the EEA, this National MA can be recognized in another Member States through the Mutual Recognition Procedure.
Where a product has already been authorized for marketing in a Member State of the EEA, this National MA can be recognized in other Member States through the Mutual Recognition Procedure.
Their utility is further limited by secondary resistance mechanisms arising in the relapsed or refractory settings, as well as the antigen heterogeneity that is characteristic of some of these diseases. • Limited Access: Due to the potential for severe toxicities, the limited number of safe and efficacious targets, supply constraints due to manufacturing complexity and scalability of processes, length of the regulatory process, and the substantial capital requirements for bringing cell therapies to market at scale, CAR-Ts are still not widely available for oncology patients.
Their utility is further limited by secondary resistance mechanisms arising in the relapsed or refractory settings, as well as the antigen heterogeneity that is characteristic of some of these diseases. • Limited Access: Due to the potential for severe toxicities, the limited number of treatment centers, supply constraints due to manufacturing complexity and scalability of processes, length of the regulatory process, and the substantial capital requirements for bringing cell therapies to market at scale, CAR-Ts are still not widely available for oncology patients.
Existing cell therapy solutions, most of which use a biologic-based, single chain variable fragment (scFv) binding domain, tend to be difficult to manufacture, beneficial to a limited segment of patients, often result in high toxicity, and have narrow applicability in treatable indications.
Existing CAR-T therapy solutions, most of which use a biologic-based, single chain variable fragment (scFv) binding domain, tend to be difficult to manufacture, beneficial to a limited segment of patients, often result in high toxicity, and have narrow applicability in treatable indications.
We initiated our Phase 1 dose-escalation clinical trial of ACLX-001 in the second quarter of 2022. This trial is intended to serve as clinical validation of our ARC-SparX platform as we seek to understand PK, safety profile, and dosing strategy for future clinical development.
We initiated our Phase 1 dose-escalation clinical trial of ACLX-001 in 2022. This trial is intended to serve as clinical validation of our ARC-SparX platform as we seek to understand PK, safety profile, and dosing strategy for future clinical development.
Carvykti, developed by Legend/Johnson & Johnson, has demonstrated an ORR of 97.9%, a CR/sCR rate of 82% and an estimated median progression-free survival (mPFS) of 34.9 months in the Phase 1b/2 CARTITUDE-1 trial in patients with rrMM that had received 3 or more prior lines of therapy.
Carvykti, developed by Legend/Johnson & Johnson, has demonstrated an ORR of 97.9%, a CR/sCR rate of 82%, an estimated median progression-free survival (mPFS) of 34.9 months, and an estimated median overall survival (mOS) of 60.7 months in the Phase 1b/2 CARTITUDE-1 trial in patients with rrMM that had received 3 or more prior lines of therapy.
Our Chief Financial Officer, Michelle Gilson, was previously a senior equity research analyst covering the biotechnology sector, most recently as a Managing Director at Canaccord Genuity. We have attracted a diverse and talented group of innovators and company builders to help us execute our strategy and to build a transformative cell therapy platform company.
Our Chief Financial Officer, Michelle Gilson, was previously a senior equity research analyst covering the biotechnology sector, most recently as a Managing Director at Canaccord Genuity. We have attracted a diverse and talented group of innovators and company builders to help us execute our strategy and to build a transformative CAR-T platform company.
Collectively, we are driven by our shared purpose and our values. As of December 31, 2024, we had 163 full-time employees and we are committed to continuing to build and maintain a diverse and inclusive organization. We believe focusing on diversity and inclusion is not only the right thing to do but is also a competitive advantage.
Collectively, we are driven by our shared purpose and our values. As of December 31, 2025, we had 209 full-time employees and we are committed to continuing to build and maintain a diverse and inclusive organization. We believe focusing on diversity and inclusion is not only the right thing to do but is also a competitive advantage.
Many benefits accrue to sponsors of product candidates with PRIME designation, including but not limited to, early and proactive regulatory dialogue with the EMA, frequent discussions on clinical trial designs and other development program elements, and accelerated marketing authorization application assessment once a dossier has been submitted.
Many benefits accrue to sponsors of product candidates with PRIME designation, including but not limited to, early and proactive regulatory dialogue with the EMA, frequent discussions on clinical trial designs and other development program elements, and the possibility of an accelerated assessment of a marketing authorization application, where applicable, once a dossier has been submitted.
MM is the third most common hematological malignancy in the United States and Europe, with approximately 35,000 new cases diagnosed per year in the United States.
MM is the third most common hematological malignancy in the United States and Europe, with approximately 36,000 new cases diagnosed per year in the United States.
The recent availability of cell therapy products introduced an unprecedented “living therapeutic” modality that offers benefits well beyond what previous oncology modalities offered. For the first time, these therapeutics directly harness the strength of the patient’s own immune system to significantly reduce, even potentially eradicate, tumors.
The recent availability of CAR-T products introduced an unprecedented “living therapeutic” modality that offers benefits well beyond what previous oncology modalities offered. For the first time, these therapeutics directly harness the strength of the patient’s own immune system to significantly reduce, even potentially eradicate, tumors.
Particularly striking is that these responses are achieved with a single, personalized administration of the cell therapy, generally achieving rapid and durable responses with toxicities resolving in days to weeks. This transformative therapy results in extended quality of life benefits without maintenance or additional treatment.
Particularly striking is that these responses are achieved with a single, personalized administration of the CAR-T, generally achieving rapid and durable responses with 10 toxicities resolving in days to weeks. This transformative therapy results in extended quality of life benefits without maintenance or additional treatment.
In MM, we plan to: • Evaluate the efficacy of our lead product candidate, anito-cel, in our pivotal Phase 2 iMMagine-1 and Phase 3 iMMagine-3 trials in rrMM and seek regulatory approval in collaboration with Kite; • In collaboration with Kite, pursue expanded access to anito-cel through other label expansion clinical trials, including iMMagine-3 and others; • Through our ex-U.S. partner, Kite, pursue clinical development of anito-cel in other key geographies, such as Europe and Asia; and • Evaluate the potential of our ARC-SparX technology through our ongoing Phase 1 clinical trial of ACLX-001 in rrMM.
In MM, we plan to: • In collaboration with Kite, seek regulatory approval of our lead product candidate, anito-cel, with results from our Phase 1 clinical trial and our pivotal Phase 2 iMMagine-1 clinical trial in rrMM; • In collaboration with Kite, pursue expanded access to anito-cel through other label expansion clinical trials, including iMMagine-3, GEM-AnitoFIRST and others; 19 • Through our ex-U.S. partner, Kite, pursue clinical development of anito-cel in other key geographies, such as Europe and Asia; and • Evaluate the potential of our ARC-SparX technology through our ongoing Phase 1 clinical trial of ACLX-001 in rrMM.
Additionally, pursuant to the Kite Collaboration Agreement, we and Kite will be jointly responsible for commercialization of anito-cel and certain other MM cell therapy products, if approved by the FDA, in the United States and plan to leverage Kite’s commercialization infrastructure, including sales and marketing and commercial distribution.
Additionally, pursuant to the Kite Collaboration Agreement, we and Kite will be jointly responsible for commercialization of anito-cel and certain other MM CAR-T products, if approved by the FDA, in the United States and plan to leverage Kite’s commercialization infrastructure, including sales and marketing and commercial distribution.
We estimate that the size of the global MM market was approximately $25 billion in 2024 and that the current total addressable global CAR-T market for rrMM to be $12 billion or more based on the number of patients who are receiving second line treatments and beyond.
We estimate that the size of the global MM market was approximately $26 billion in 2025 and that the current total addressable global CAR-T market for rrMM to be $12 billion or more based on the number of patients who are receiving second line treatments and beyond.
We estimate that the size of the global MM market was approximately $25 billion in 2024 and that the current total addressable CAR-T market for rrMM to be $12 billion or more based on the number of patients who are receiving second line treatments and beyond.
We estimate that the size of the global MM market was approximately $26 billion in 2025 and that the current total addressable CAR-T market for rrMM to be $12 billion or more based on the number of patients who are receiving second line treatments and beyond.
This followed the completion of the technical transfer to Kite, which was announced in May 2024, as well as the transfer of the Investigational New Drug (IND) application for anito-cel in rrMM, which has been cleared by the U.S. Food and Drug Administration (FDA).
This follows the completion of the technical transfer to Kite, which was announced in May 2024, as well as the transfer of the Investigational New Drug (IND) application for anito-cel in MM, which has been cleared by the U.S. Food and Drug Administration (FDA).
As the field of adoptive cell therapy looks to apply the technology beyond oncology, including transplantation, autoimmune, cardiac, infectious and neurological diseases, so too do we seek to explore such opportunities. We envision expanding into treatments for antibody-mediated autoimmune diseases.
As the field of adoptive CAR-T looks to apply the technology beyond oncology, including transplantation, autoimmune, cardiac, infectious and neurological diseases, so too do we seek to explore such opportunities. We envision expanding into treatments for antibody-mediated autoimmune diseases.
Our Team Our team and culture are critical to realizing our vision of reimagining cell therapy as one of the future pillars of medicine. We are led by a diverse team of executives with significant experience in business, discovery, development, manufacturing, and commercialization of differentiated and novel therapies specifically in the fields of oncology, cell therapy and rare diseases.
Our Team Our team and culture are critical to realizing our vision of reimagining immunotherapies as one of the future pillars of medicine. We are led by a diverse team of executives with significant experience in business, discovery, development, manufacturing, and commercialization of differentiated and novel therapies specifically in the fields of oncology, CAR-T and rare diseases.
We believe it may be important for patients to have both autologous and allogeneic/off-the-shelf cell therapy options in certain settings as both therapeutic options mature, including therapies derived from T-cells and NK cells.
We believe it may be important for patients to have both autologous and off-the-shelf CAR-T options in certain settings as both therapeutic options mature, including therapies derived from T-cells and NK cells.
Our Pipeline We have built a broad and scalable pipeline that has positioned us to capitalize on the potential of our proprietary platform technologies and achieve long-term growth and sustainability within the field of cell therapy.
Our Pipeline We have built a broad and scalable pipeline that has positioned us to capitalize on the potential of our proprietary platform technologies and achieve long-term growth and sustainability within the field of CAR-T.
Initially evaluated in indications where patients were refractory to multiple lines of therapy and had generally exhausted their therapeutic options, adoptive cell therapies have shown response rates that exceed many other available modalities, and are now being evaluated in earlier line settings.
Initially evaluated in indications where patients were refractory to multiple lines of therapy and had generally exhausted their therapeutic options, CAR-Ts have shown response rates that exceed many other available modalities, and are now being evaluated in earlier line settings.
Under the Kite Collaboration Agreement, as further described in “Licenses and Collaborations” below, Kite will develop allogeneic/off-the-shelf cell therapies for the treatment of myeloma as another tool in our fight against cancer that includes our autologous ddCARs and ARC-SparX. Indications Beyond Oncology .
Under the Kite Collaboration Agreement, as further described in “Licenses and Collaborations” below, Kite will develop next generation cell therapies for the treatment of myeloma as another tool in our fight against cancer that includes our autologous ddCARs and ARC-SparX. Indications Beyond Oncology .
The aggressive growth of AML in the bone marrow and blood, its disruption of normal blood cell production and the lack of durable treatments leave AML patients with an estimated 35% five-year survival rate. According to the National Cancer Institute SEER database, there were estimated to be 73,168 people living with AML in the USA in 2020.
The aggressive growth of AML in the bone marrow and blood, its disruption of normal blood cell production and the lack of durable treatments leave AML patients with an estimated 35% five-year survival rate. According to the National Cancer Institute SEER database, there were estimated to be 79,317 people living with AML in the USA in 2022.
Additionally, Carvykti has a Black Box warning for Parkinsonism and Guillain-Barré syndrome, and its label contains Warnings and Precautions for Neurologic Toxicities, including those not considered ICANS such as Parkinsonism, Guillain-Barré Syndrome, Immune Mediated Myelitis, Peripheral Neuropathy, and Cranial Nerve Palsies.
For example, Carvykti has a Black Box warning for Parkinsonism, Guillain-Barré syndrome, and IEC-EC, and its label contains Warnings and Precautions for 21 Neurologic Toxicities, including those not considered ICANS such as Parkinsonism, Guillain-Barré Syndrome, Immune Mediated Myelitis, Peripheral Neuropathy, Cranial Nerve Palsies, and IEC-EC.
From our clinical trials of anito-cel, we reported preliminary data that we believe supports efficacy and safety benefits, as well as potential manufacturability advantages associated with our D-Domain technology. The recent availability of cell therapy products, such as CAR-T-cells, introduced an unprecedented “living therapeutic” modality that offers benefits well beyond what previous oncology modalities offered.
From our clinical trials of anito-cel, we reported preliminary data that we believe supports efficacy and safety benefits, as well as potential manufacturability advantages associated with our D-Domain technology. 6 The recent availability of CAR-T products introduces an unprecedented “living therapeutic” modality that offers benefits well beyond what previous oncology modalities offered.
The median time to onset of CRS was 4 days with a median duration of 3 days. 98% of patients had CRS that either resolved within 14 days of anito-cel infusion or did not experience any CRS. Any grade ICANS was observed in 9 patients (9%) with 4 (4%) Grade 1, 4 (4%) Grade 2, and 1 (1%) Grade 3.
The median time to onset of CRS was 4 days with a median duration of 2 days. 95% of patients had CRS that either resolved within 10 days of anito-cel infusion or did not experience any CRS. Any grade ICANS was observed in 9 patients (8%) with 3 (3%) Grade 1, 5 (4%) Grade 2, and 1 (1%) Grade 3.
We believe further implementation of AI and ML can assist in other areas of the discovery process such as D-Domain affinity optimization from deep learning of analysis of thousands of D-Domain sequences from our panning and screening campaigns. Next-Generation Cell Therapy Products, such as Allogeneic and Other Immune Cell Therapies .
We believe further implementation of AI and ML can assist in other areas of the discovery process such as D-Domain affinity optimization from deep learning of analysis of thousands of D-Domain sequences from our panning and screening campaigns. Next-Generation Immunotherapy Products .
This family includes 21 issued patents (including 3 issued U.S. patents) and 20 pending applications. Specifically, issued/granted claims encompass anito-cel and universal ARC-T-cells, ACLX-001: BCMA and ACLX-002: CD123 SparXs, and methods of use thereof in the treatment of cancer.
This family includes 25 issued patents (including 4 issued U.S. patents) and 16 pending applications. Specifically, issued/granted claims encompass anito-cel and universal ARC-T-cells, ACLX-001: BCMA and ACLX-002: CD123 SparXs, and methods of use thereof in the treatment of cancer.
According to the FDA’s FY 2025 fee schedule, effective through September 30, 2025, the user fee for an application requiring clinical data, such as a BLA, is approximately $4.3 million. PDUFA also imposes an annual program fee for each marketed human biologic ($403,889 in FY 2025) and an annual establishment fee on facilities used to manufacture prescription biologics.
According to the FDA’s FY 2026 fee schedule, effective through September 30, 2026, the user fee for an application requiring clinical data, such as a BLA, is approximately $4.68 million. PDUFA also imposes an annual program fee for each marketed human biologic ($442,213 in FY 2026) and an annual establishment fee on facilities used to manufacture prescription biologics.
We believe our D-Domain technology is a transformational platform that enables us to take the right approach for the right indication within cell therapy.
We believe our D-Domain technology is a transformational platform that enables us to take the right approach for the right indication within CAR-T.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeFuture growth would impose significant added responsibilities on members of management, including: • Identifying, recruiting, integrating, retaining and motivating additional employees and other service providers; • Managing our internal development efforts effectively, including the clinical and FDA review process for our product candidates, while complying with our contractual obligations to contractors and other third parties; and • Improving our operational, financial and management controls, reporting systems and procedures. 68 Our future financial performance and our ability to commercialize our product candidates will depend, in part, on our ability to effectively manage any future growth, and our management may have to divert a disproportionate amount of its attention away from day-to-day activities in order to devote a substantial amount of time to managing these growth activities, or spend additional resources to add those capabilities or outsource them.
Biggest changeFuture growth would impose significant added responsibilities on members of management, including: • Identifying, recruiting, integrating, retaining and motivating additional employees and other service providers; • Managing our internal development efforts effectively, including the clinical and FDA review process for our product candidates, while complying with our contractual obligations to contractors and other third parties; and • Improving our operational, financial and management controls, reporting systems and procedures.
Although the FDA has previously issued and lifted one partial clinical hold, there is no assurance that the FDA will not issue another clinical hold in the future.
Although the FDA has previously issued and lifted one partial clinical hold, there is no assurance that the FDA will not issue another clinical hold in the future.
To the extent the FDA issues another clinical hold, addressing a clinical hold takes considerable time and expense and there can be no assurance that the FDA will remove a clinical hold in a timely manner, or at all, in which case our business and prospects for development and approval of anito-cel would be materially harmed.
To the extent the FDA issues another clinical hold, addressing a clinical hold takes considerable time and expense and there can be no assurance that the FDA will remove a clinical hold in a timely manner, or at all, in which case our business and prospects for development and approval of anito-cel would be materially harmed.
We do not have any product candidates approved for sale in any jurisdiction, including international markets, and we do not have experience in obtaining regulatory approval in international markets.
We do not have any product candidates approved for sale in any jurisdiction, including international markets, and we do not have experience in obtaining regulatory approval in international markets.
Disruptions at the FDA and other foreign, federal, state, or local regulatory agencies, such as furloughs or government shutdowns, restrictions due to the COVID-19 pandemic or other public health concerns, travel restrictions, or staffing shortages, may also increase the time necessary for new drugs to be reviewed and/or approved by necessary government agencies, which would adversely affect our business.
Disruptions at the FDA and other foreign, federal, state, or local regulatory agencies, such as furloughs or government shutdowns, restrictions due to public health concerns such as the COVID-19 pandemic, travel restrictions, or staffing shortages, may also increase the time necessary for new drugs to be reviewed and/or approved by necessary government agencies, which would adversely affect our business.
Events that may prevent successful or timely completion of clinical development include: • Delays associated with events out of our control such as global pandemics or geopolitical uncertainty and instability, as further described under Risks Related to Our Business; • Delays in reaching a consensus with regulatory agencies on trial design; • Delays in reaching agreement on acceptable terms with prospective CROs, and clinical trial sites and obtaining required institutional review board (IRB), approval at each clinical trial site; • Delays in recruiting and enrolling suitable patients to participate in our clinical trials; • Failure to collect sufficiently viable white blood cells from patients, adequately expand or successfully transduce sufficient number of patient T-cells for infusion or otherwise manufacture product candidates, or infuse patients in a timely manner with product candidate; • Failure by our CROs, other third parties or us to adhere the trial protocol or the FDA’s good clinical practices (GCPs) or applicable regulatory guidelines in other countries; • Third-party contractors becoming debarred or suspended or otherwise penalized by the FDA or foreign health authorities for violations of applicable regulatory requirements; • Delays in the testing, validation, manufacturing and delivery of our product candidates to the clinical trial sites, including due to a facility manufacturing any of our product candidates or any of their components being ordered by the FDA or foreign health authorities to temporarily or permanently shut down due to violations of current good manufacturing practices (cGMPs) regulations or other applicable requirements, or infections or cross-contaminations of product candidates in the manufacturing process; • Delays in the technology transfer and scale up of our manufacturing process to support late-stage clinical trials; • Delays in having patients complete their participation in a trial or return for post-treatment follow-up visits; • Clinical trial sites or patients dropping out of a trial or experiencing changing health or other conditions that require removing them from the trial; • Discovering that product candidates have unforeseen safety issues, undesirable side effects or other unexpected characteristics; • To the extent that we conduct clinical trials in foreign countries, the failure of enrolled patients in foreign countries to adhere to clinical protocol as a result of differences in healthcare services or cultural customs, managing additional administrative burdens associated with foreign regulatory schemes, as well as political and economic risks relevant to such foreign countries; • Receiving untimely or unfavorable feedback from applicable regulatory authorities regarding the trial or requests from regulatory authorities to modify the design of a trial; 59 • Suspensions or terminations by IRBs or Data Safety Monitoring Boards (DSMBs) or internal clinical holds and/or clinical holds from or by regulatory authorities; • Lack of adequate funding to continue operations; or • Changes in regulatory requirements and guidance that require amending or submitting new clinical protocols and/or amendments to INDs.
Events that may prevent successful or timely completion of clinical development include: • Delays associated with events out of our control such as global pandemics or geopolitical uncertainty and instability, as further described under Risks Related to Our Business; • Delays in reaching a consensus with regulatory agencies on trial design; • Delays in reaching agreement on acceptable terms with prospective CROs, and clinical trial sites and obtaining required institutional review board (IRB), approval at each clinical trial site; • Delays in recruiting and enrolling suitable patients to participate in our clinical trials; • Failure to collect sufficiently viable white blood cells from patients, adequately expand or successfully transduce sufficient number of patient T-cells for infusion or otherwise manufacture product candidates, or infuse patients in a timely manner with product candidate; • Failure by our CROs, other third parties or us to adhere the trial protocol or the FDA’s good clinical practices (GCPs) or applicable regulatory guidelines in other countries; • Third-party contractors becoming debarred or suspended or otherwise penalized by the FDA or foreign health authorities for violations of applicable regulatory requirements; • Delays in the testing, validation, manufacturing and delivery of our product candidates to the clinical trial sites, including due to a facility manufacturing any of our product candidates or any of their components being ordered by the FDA or foreign health authorities to temporarily or permanently shut down due to violations of current good manufacturing practices (cGMPs) regulations or other applicable requirements, or infections or cross-contaminations of product candidates in the manufacturing process; • Delays in the technology transfer and scale up of our manufacturing process to support late-stage clinical trials; • Delays in having patients complete their participation in a trial or return for post-treatment follow-up visits; • Clinical trial sites or patients dropping out of a trial or experiencing changing health or other conditions that require removing them from the trial; • Discovering that product candidates have unforeseen safety issues, undesirable side effects or other unexpected characteristics; • To the extent that we conduct clinical trials in foreign countries, the failure of enrolled patients in foreign countries to adhere to clinical protocol as a result of differences in healthcare services or cultural customs, managing additional administrative burdens associated with foreign regulatory schemes, as well as political and economic risks relevant to such foreign countries; • Receiving untimely or unfavorable feedback from applicable regulatory authorities regarding the trial or requests from regulatory authorities to modify the design of a trial; • Suspensions or terminations by IRBs or Data Safety Monitoring Boards (DSMBs) or internal clinical holds and/or clinical holds from or by regulatory authorities; • Lack of adequate funding to continue operations; or • Changes in regulatory requirements and guidance that require amending or submitting new clinical protocols and/or amendments to INDs.
The criminal FCA provides for criminal penalties for submitting false claims, including imprisonment and criminal fines. • The Civil Monetary Penalty Act of 1981 and implementing regulations, which impose penalties against any person or entity that, among other things, is determined to have presented or caused to be presented a claim to a federal healthcare program that the person knows or should know is for an item or service that was not provided as claimed or is false or fraudulent, or offered or transferred remuneration to a federal healthcare beneficiary that a person knows or should know is likely to influence the beneficiary’s decision to order or receive items or services reimbursable by the government from a particular provider or supplier. • The federal Health Insurance Portability and Accountability Act of 1996 (HIPAA), which created new federal criminal statutes that prohibit knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless of the payor (e.g., public or private) and knowingly and willfully falsifying, concealing or covering up by any trick or device a material fact or making any materially false statements in connection with the delivery of, or payment for, healthcare benefits, items or services relating to healthcare matters. • HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (HITECH), and their respective implementing regulations, which impose requirements on certain covered healthcare providers, health plans, and healthcare clearinghouses as well as their respective business associates that perform services for them that involve the use, or disclosure of, individually identifiable health information, relating to the privacy, security and transmission of individually identifiable health information without appropriate authorization. • The federal Physician Payment Sunshine Act, created under the Patient Protection and Affordable Care Act as amended by the Health Care and Education Reconciliation Act of 2010, collectively, the Affordable Care Act (ACA), and its implementing regulations, which require applicable manufacturers of covered drugs, devices, biologicals and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to the Centers for Medicare & Medicaid Services (CMS) of the U.S.
The criminal FCA provides for criminal penalties for submitting false claims, including imprisonment and criminal fines. • The Civil Monetary Penalty Act of 1981 and implementing regulations, which impose penalties against any person or entity that, among other things, is determined to have presented or caused to be presented a claim to a federal healthcare program that the person knows or should know is for an item or service that was not provided as claimed or is false or fraudulent, or offered or transferred remuneration to a federal healthcare beneficiary that a person knows or should know 87 is likely to influence the beneficiary’s decision to order or receive items or services reimbursable by the government from a particular provider or supplier. • The federal Health Insurance Portability and Accountability Act of 1996 (HIPAA), which created new federal criminal statutes that prohibit knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless of the payor (e.g., public or private) and knowingly and willfully falsifying, concealing or covering up by any trick or device a material fact or making any materially false statements in connection with the delivery of, or payment for, healthcare benefits, items or services relating to healthcare matters. • HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (HITECH), and their respective implementing regulations, which impose requirements on certain covered healthcare providers, health plans, and healthcare clearinghouses as well as their respective business associates that perform services for them that involve the use, or disclosure of, individually identifiable health information, relating to the privacy, security and transmission of individually identifiable health information without appropriate authorization. • The federal Physician Payment Sunshine Act, created under the Patient Protection and Affordable Care Act as amended by the Health Care and Education Reconciliation Act of 2010, collectively, the Affordable Care Act (ACA), and its implementing regulations, which require applicable manufacturers of covered drugs, devices, biologicals and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to the Centers for Medicare & Medicaid Services (CMS) of the U.S.
We plan to seek regulatory approval of our product candidates outside of the United States and, accordingly, we expect that we will be subject to additional risks related to operating in foreign countries if we obtain the necessary approvals, including: • Differing regulatory requirements in foreign countries, including constraints on manufacturing; • Additional trials in foreign countries; • Requirement to secure and validate region-specific manufacturing and clinical and commercial supply; • Unexpected changes in tariffs, trade barriers, price and exchange controls and other regulatory requirements; • Economic weakness, including inflation, or political instability in particular foreign economies and markets; • Compliance with tax, employment, immigration and labor laws for employees living or traveling abroad; • Foreign taxes, including withholding of payroll taxes; • Foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country; • Difficulties staffing and managing foreign operations; • Workforce uncertainty in countries where labor unrest is more common than in the United States; • Potential liability under the Foreign Corrupt Practices Act of 1977 or comparable foreign regulations; • Challenges enforcing our contractual and intellectual property rights, especially in those foreign countries that do not respect and protect intellectual property rights to the same extent as the United States; • Production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and 101 • Business interruptions resulting from geo-political actions, including war (including ongoing geopolitical tensions related to the Russia and Ukraine conflict, resulting sanctions imposed by the United States and other countries and retaliatory actions taken by Russia in response to such sanctions, and the war in the Middle East), armed conflict, terrorist activities, global pandemics and terrorism.
We plan to seek regulatory approval of our product candidates outside of the United States and, accordingly, we expect that we will be subject to additional risks related to operating in foreign countries if we obtain the necessary approvals, including: • Differing regulatory requirements in foreign countries, including constraints on manufacturing; • Additional trials in foreign countries; • Requirement to secure and validate region-specific manufacturing and clinical and commercial supply; • Unexpected changes in tariffs, trade barriers, price and exchange controls and other regulatory requirements; • Economic weakness, including inflation, or political instability in particular foreign economies and markets; • Compliance with tax, employment, immigration and labor laws for employees living or traveling abroad; • Foreign taxes, including withholding of payroll taxes; • Foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country; • Difficulties staffing and managing foreign operations; • Workforce uncertainty in countries where labor unrest is more common than in the United States; • Potential liability under the Foreign Corrupt Practices Act of 1977 or comparable foreign regulations; • Challenges enforcing our contractual and intellectual property rights, especially in those foreign countries that do not respect and protect intellectual property rights to the same extent as the United States; • Production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and • Business interruptions resulting from geo-political actions, including war (including ongoing geopolitical tensions related to the Russia and Ukraine conflict, resulting sanctions imposed by the United States and other countries and retaliatory actions taken by Russia in response to such sanctions, and the war in the Middle East), armed conflict, terrorist activities, global pandemics and terrorism.
Our future capital requirements will depend on many factors, including: • The scope, progress, timing, results and costs of developing and manufacturing our product candidates, and their components, and conducting preclinical studies and clinical trials and other testing of our product candidates; • Our ability to continue our business operations and product candidate research and development, and to adapt to any changes in the regulatory approval process, manufacturing supply, or clinical trial requirements and timing; • The costs, timing and outcome of regulatory review of any of our product candidates; • The costs and timing of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending any intellectual property-related claims, including any claims by third parties that we are infringing upon their intellectual property rights; • Our ability to establish and maintain strategic collaborations, licensing or other arrangements and the financial terms of any such agreements, including the timing and amount of any future milestone, royalty or other payments due under any such agreement; • The costs and timing of future commercialization activities, including manufacturing, marketing, sales and distribution, for any of our product candidates for which we receive marketing approval; • The extent to which our product candidates, if approved, can be offered by prescribers in various clinical settings, including academic hospitals and community practices, the acceptance of our products, if and when approved, by patients, the medical community and third-party payors, and the revenue received from commercial sale of any products for which we receive marketing approval; • The effect of competing technologies and market developments; and • The extent to which we acquire or invest in other businesses, products and technologies and any other licensing or collaboration arrangements for any of our product candidates.
Our future capital requirements will depend on many factors, including: • The scope, progress, timing, results and costs of developing and manufacturing our product candidates, and their components, and conducting preclinical studies and clinical trials and other testing of our product candidates; • Our ability to continue our business operations and product candidate research and development, and to adapt to any changes in the regulatory approval process, manufacturing supply, or clinical trial requirements and timing; • The costs, timing and outcome of regulatory review of any of our product candidates; • The costs and timing of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending any intellectual property-related claims, including any claims by third parties that we are infringing upon their intellectual property rights; • Our ability to establish and maintain strategic collaborations, licensing or other arrangements and the financial terms of any such agreements, including the timing and amount of any future milestone, royalty or other payments due under any such agreement; • The costs and timing of future commercialization activities, including manufacturing, marketing, sales and distribution, for any of our product candidates for which we receive marketing approval; 57 • The extent to which our product candidates, if approved, can be offered by prescribers in various clinical settings, including academic hospitals and community practices, the acceptance of our products, if and when approved, by patients, the medical community and third-party payors, and the revenue received from commercial sale of any products for which we receive marketing approval; • The effect of competing technologies and market developments; and • The extent to which we acquire or invest in other businesses, products and technologies and any other licensing or collaboration arrangements for any of our product candidates.
The degree of market acceptance of our product candidates, if approved for commercial sale, will depend on a number of factors, including: • The clinical indications for which our product candidates are approved; • The willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; • Physicians, hospitals, cancer treatment centers and patients considering our product candidates as a safe, pure and effective treatment; • The potential and perceived advantages of our product candidates over alternative treatments; • Our ability to demonstrate the advantages of our product candidates over other conventional CAR-T therapies; • The perceived prevalence and severity of any side effects for our product candidates compared to the prevalence and severity of any side effects for conventional CAR-T products and other cell therapies; • Product labeling, limitations, warnings or product insert requirements of the FDA or foreign health authorities, including FDA’s requirement for manufacturers of approved CAR-T therapies to include boxed warning regarding T cell malignancies; • The timing of market introduction of our product candidates as well as competitive products; • The cost of treatment in relation to alternative treatments; • Factors that impact access to apheresis, including but not limited to availability of facilities that offer apheresis, additional costs associated with apheresis and administration of CAR-T therapies, and other hospital infrastructure and bottlenecks; • The availability of adequate coverage, reimbursement and pricing by third-party payors and government authorities; • The willingness of patients to pay out-of-pocket in the absence of coverage by third-party payors and government authorities; • Relative convenience and ease of administration, including as compared to alternative treatments and competitive therapies; and • The effectiveness of our sales and marketing efforts.
The degree of market acceptance of our product candidates, if approved for commercial sale, will depend on a number of factors, including: • The clinical indications for which our product candidates are approved; • The willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; • Physicians, hospitals, cancer treatment centers and patients considering our product candidates as a safe, pure and effective treatment; • The potential and perceived advantages of our product candidates over alternative treatments; • Our ability to demonstrate the advantages of our product candidates over other conventional CAR-T therapies; • The perceived prevalence and severity of any side effects for our product candidates compared to the prevalence and severity of any side effects for conventional CAR-T products and other cell therapies; 70 • Product labeling, limitations, warnings or product insert requirements of the FDA or foreign health authorities, including FDA’s requirement for manufacturers of approved CAR-T therapies to include boxed warning regarding T cell malignancies; • The timing of market introduction of our product candidates as well as competitive products; • The cost of treatment in relation to alternative treatments; • Factors that impact access to apheresis, including but not limited to availability of facilities that offer apheresis, additional costs associated with apheresis and administration of CAR-T therapies, and other hospital infrastructure and bottlenecks; • The availability of adequate coverage, reimbursement and pricing by third-party payors and government authorities; • The willingness of patients to pay out-of-pocket in the absence of coverage by third-party payors and government authorities; • Relative convenience and ease of administration, including as compared to alternative treatments and competitive therapies; and • The effectiveness of our sales and marketing efforts.
In such an event, competitors might be able to enter the market earlier than would otherwise have been the case; • The coverage claimed in a patent application can be significantly reduced before the patent is issued, and its scope can be reinterpreted after issuance; • Patent applications may not result in any patents being issued; • Patents that may be issued or in-licensed may be challenged, invalidated, modified, revoked, circumvented, narrowed, found to be unenforceable or otherwise may not provide any competitive advantage; • Our competitors, many of whom have substantially greater resources and many of whom have made significant investments in competing technologies, may seek or may have already obtained patents that will limit, interfere with or eliminate our ability to make, use, and sell our potential product candidates; • There may be significant pressure on the U.S. government and international governmental bodies to limit the scope of patent protection both in the United States and abroad for disease treatments that prove successful, as a matter of public policy regarding worldwide health concerns; and • Countries other than the United States may have patent laws less favorable to patentees than those upheld by U.S. courts, allowing foreign competitors a better opportunity to create, develop and market competing product candidates.
In such an event, competitors might be able to enter the market earlier than would otherwise have been the case; • The coverage claimed in a patent application can be significantly reduced before the patent is issued, and its scope can be reinterpreted after issuance; 98 • Patent applications may not result in any patents being issued; • Patents that may be issued or in-licensed may be challenged, invalidated, modified, revoked, circumvented, narrowed, found to be unenforceable or otherwise may not provide any competitive advantage; • Our competitors, many of whom have substantially greater resources and many of whom have made significant investments in competing technologies, may seek or may have already obtained patents that will limit, interfere with or eliminate our ability to make, use, and sell our potential product candidates; • There may be significant pressure on the U.S. government and international governmental bodies to limit the scope of patent protection both in the United States and abroad for disease treatments that prove successful, as a matter of public policy regarding worldwide health concerns; and • Countries other than the United States may have patent laws less favorable to patentees than those upheld by U.S. courts, allowing foreign competitors a better opportunity to create, develop and market competing product candidates.
The laws that may affect our ability to operate include, but are not limited to the following: • The federal Anti-Kickback Statute, which prohibits, among other things, knowingly and willfully soliciting, receiving, offering or paying any remuneration (including any kickback, bribe, or rebate), directly or indirectly, overtly or covertly, in cash or in kind, to induce, or in return for, either the referral of an individual, or the purchase, lease, order or recommendation of any good, facility, item or service for which payment may be made, in whole or in part, under a federal healthcare program, such as the Medicare and Medicaid programs. 70 • The federal civil and criminal false claims laws, including the civil False Claims Act (FCA), that can be enforced by private citizens through civil whistleblower or qui tam actions, prohibit individuals or entities from, among other things, knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government.
The laws that may affect our ability to operate include, but are not limited to the following: • The federal Anti-Kickback Statute, which prohibits, among other things, knowingly and willfully soliciting, receiving, offering or paying any remuneration (including any kickback, bribe, or rebate), directly or indirectly, overtly or covertly, in cash or in kind, to induce, or in return for, either the referral of an individual, or the purchase, lease, order or recommendation of any good, facility, item or service for which payment may be made, in whole or in part, under a federal healthcare program, such as the Medicare and Medicaid programs. • The federal civil and criminal false claims laws, including the civil False Claims Act (FCA), that can be enforced by private citizens through civil whistleblower or qui tam actions, prohibit individuals or entities from, among other things, knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government.
In addition, a new manufacturer would have to be educated in, or develop substantially equivalent processes for, the production of our product candidates. 76 • Third party manufacturers may have little or no experience with our product candidates, and therefore may require a significant amount of support from us in order to implement and maintain the infrastructure and processes required to manufacture our product candidates. • Third party manufacturers might be unable to timely manufacture our product candidates or produce the quantity and quality required to meet our clinical and commercial needs, if any. • Third party manufacturers may not be able to execute our manufacturing procedures and other logistical support requirements appropriately. • Third party manufacturers may not perform as agreed, may not devote sufficient resources to our product candidates or may not remain in the contract manufacturing business for the time required to supply our clinical trials or to successfully produce, store, and distribute our products, if any. • Manufacturers are subject to ongoing periodic unannounced inspection by the FDA and corresponding state agencies to ensure strict compliance with cGMP and other government regulations and corresponding foreign standards.
In addition, a new manufacturer would have to be educated in, or develop substantially equivalent processes for, the production of our product candidates. • Third-party manufacturers may have little or no experience with our product candidates, and therefore may require a significant amount of support from us in order to implement and maintain the infrastructure and processes required to manufacture our product candidates. • Third-party manufacturers might be unable to timely manufacture our product candidates or produce the quantity and quality required to meet our clinical and commercial needs, if any. • Third-party manufacturers may not be able to execute our manufacturing procedures and other logistical support requirements appropriately. • Third-party manufacturers may not perform as agreed, may not devote sufficient resources to our product candidates or may not remain in the contract manufacturing business for the time required to supply our clinical trials or to successfully produce, store, and distribute our products, if any. • Manufacturers are subject to ongoing periodic unannounced inspection by the FDA and corresponding state agencies to ensure strict compliance with cGMP and other government regulations and corresponding foreign standards.
Any future collaborations we might enter into may pose a number of risks, including the following: • Collaborators may not perform their obligations as expected; • Collaborators may not pursue development and commercialization of product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus or available funding or external factors, such as an acquisition, that divert resources or create competing priorities; 79 • Collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing; • Collaborators could fail to make timely regulatory submissions for a product candidate; • Collaborators may not comply with all applicable regulatory requirements or may fail to report safety data in accordance with all applicable regulatory requirements, which could subject them or us to regulatory enforcement actions; • Collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; • Product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; • A collaborator with marketing and distribution rights to one or more of our product candidates that achieve regulatory approval may not commit sufficient resources to the marketing and distribution of such product candidate or product; • Disagreements with collaborators, including disagreements over proprietary rights, contract interpretation or the preferred course of development, might cause delays or termination of the research, development or commercialization of product candidates, might lead to additional responsibilities for us with respect to product candidates, or might result in litigation or arbitration, any of which would be time consuming and expensive; • Collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation; and • Collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability.
Any future collaborations we might enter into may pose a number of risks, including the following: • Collaborators may not perform their obligations as expected; • Collaborators may not pursue development and commercialization of product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus or available funding or external factors, such as an acquisition, that divert resources or create competing priorities; • Collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing; • Collaborators could fail to make timely regulatory submissions for a product candidate; • Collaborators may not comply with all applicable regulatory requirements or may fail to report safety data in accordance with all applicable regulatory requirements, which could subject them or us to regulatory enforcement actions; • Collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; • Product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; • A collaborator with marketing and distribution rights to one or more of our product candidates that achieve regulatory approval may not commit sufficient resources to the marketing and distribution of such product candidate or product; 96 • Disagreements with collaborators, including disagreements over proprietary rights, contract interpretation or the preferred course of development, might cause delays or termination of the research, development or commercialization of product candidates, might lead to additional responsibilities for us with respect to product candidates, or might result in litigation or arbitration, any of which would be time consuming and expensive; • Collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation; and • Collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability.
Our clinical trials may fail to demonstrate adequate safety and/or efficacy of any of our product candidates. • We may encounter substantial delays, including difficulties enrolling patients, in our clinical trials. • Our product candidates may cause undesirable side effects or have other properties that could halt their clinical development, prevent their regulatory approval, require expansion of the trial size, limit their commercial potential, or result in significant negative consequences. • Interim, preliminary or topline data from our clinical trials that we announce or publish from time to time may change as more patient data become available. 50 • Manufacturing genetically engineered products is complex and subject to both human and systemic risks.
Our clinical trials may fail to demonstrate adequate safety and/or efficacy of any of our product candidates. • We may encounter substantial delays, including difficulties enrolling patients, in our clinical trials. • Our product candidates may cause undesirable side effects or have other properties that could halt their clinical development, prevent their regulatory approval, require expansion of the trial size, limit their commercial potential, or result in significant negative consequences. • Interim, preliminary or topline data from our clinical trials that we announce or publish from time to time may change as more patient data become available. • Manufacturing genetically engineered products is complex and subject to both human and systemic risks.
In August 2022, Congress passed the Inflation Reduction Act of 2022, which includes prescription drug provisions that have significant implications for the pharmaceutical industry and Medicare beneficiaries, including allowing the federal government to negotiate a maximum fair price for certain high-priced single source Medicare drugs, imposing penalties and excise tax for manufacturers that fail to comply with the drug price negotiation requirements, requiring inflation rebates for all Medicare Part B and Part D drugs, with limited exceptions, if their drug prices increase faster than inflation, and redesigning Medicare Part D to reduce out-of-pocket prescription drug costs for beneficiaries, among other changes.
In August 2022, Congress passed the Inflation Reduction Act of 2022, which includes prescription drug provisions that 72 have significant implications for the pharmaceutical industry and Medicare beneficiaries, including allowing the federal government to negotiate a maximum fair price for certain high-priced single source Medicare drugs, imposing penalties and excise tax for manufacturers that fail to comply with the drug price negotiation requirements, requiring inflation rebates for all Medicare Part B and Part D drugs, with limited exceptions, if their drug prices increase faster than inflation, and redesigning Medicare Part D to reduce out-of-pocket prescription drug costs for beneficiaries, among other changes.
Regardless of the merits or eventual outcome, liability claims may result in: • Decreased demand for our product candidates or products that we may develop; • Impairment of our business reputation; • Withdrawal of clinical trial participants; • Initiation of investigations by regulators; • Costs to defend the related litigation; • A diversion of management’s time and our resources; • Substantial monetary awards to trial participants or patients; 72 • Product recalls, withdrawals or labeling, marketing or promotional restrictions; • Loss of revenue; • Exhaustion of any available insurance and our capital resources; • The inability to commercialize any product candidate; and • A decline in our share price.
Regardless of the merits or eventual outcome, liability claims may result in: • Decreased demand for our product candidates or products that we may develop; • Impairment of our business reputation; • Withdrawal of clinical trial participants; • Initiation of investigations by regulators; • Costs to defend the related litigation; • A diversion of management’s time and our resources; • Substantial monetary awards to trial participants or patients; • Product recalls, withdrawals or labeling, marketing or promotional restrictions; • Loss of revenue; • Exhaustion of any available insurance and our capital resources; • The inability to commercialize any product candidate; and • A decline in our share price.
If a product that has orphan drug designation from the FDA subsequently receives the first FDA approval for a particular active ingredient for the disease for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications, including a BLA, to market the same biologic for the same indication, for seven years, except in limited circumstances such as a showing of clinical superiority to the product with orphan product exclusivity or if FDA finds that the holder of the orphan exclusivity has not shown that it can ensure the availability of sufficient quantities of the orphan product to meet the needs of patients with the disease or condition for which the product was designated.
If a product that has orphan drug designation from the FDA subsequently receives the first FDA approval for a particular active ingredient for the disease for which it has such designation, the product is entitled to orphan 79 product exclusivity, which means that the FDA may not approve any other applications, including a BLA, to market the same biologic for the same indication, for seven years, except in limited circumstances such as a showing of clinical superiority to the product with orphan product exclusivity or if FDA finds that the holder of the orphan exclusivity has not shown that it can ensure the availability of sufficient quantities of the orphan product to meet the needs of patients with the disease or condition for which the product was designated.
The number of shares of our common stock available for sale under our 2022 ESPP is cumulatively increased on the first day of each fiscal year, beginning with the fiscal year following the fiscal year in which the first enrollment date (if any) occurs under the 2022 ESPP, which occurred in the Company’s 2022 fiscal year, and ending on the twenty year anniversary of the date our board of directors approved the 2022 ESPP equal to the least of: (i) 312,500 shares, (ii) 1.0% of the total number of shares of our common stock outstanding as of the last day of the immediately preceding fiscal year, or (iii) a number of shares determined by the administrator of the 2022 ESPP.
The number of shares of our common stock available for sale under our 2022 ESPP is cumulatively increased on the first day of each fiscal year, beginning with the fiscal year following the fiscal year in which the first enrollment date (if any) occurs under the 2022 ESPP, which occurred in 107 the Company’s 2022 fiscal year, and ending on the twenty year anniversary of the date our board of directors approved the 2022 ESPP equal to the least of: (i) 312,500 shares, (ii) 1.0% of the total number of shares of our common stock outstanding as of the last day of the immediately preceding fiscal year, or (iii) a number of shares determined by the administrator of the 2022 ESPP.
Because of the clinical stage of development of our product candidates, our ability to eventually generate significant revenues from product sales will depend on a number of factors, including: • Identification of additional target antigens for desired indications; • Identification and development of D-Domain-based binding regions that bind to the desired target antigens; • Successful completion of preclinical studies; • Submission of INDs or other regulatory applications for our planned clinical trials or future clinical trials and authorizations from regulators to initiate clinical trials; • Successful enrollment in, and completion of, clinical trials; • Achieving favorable results from clinical trials; • Receipt of marketing approvals from applicable regulatory authorities; • Establishing and maintaining sufficient manufacturing capabilities, whether internally or with third parties, for clinical and commercial supply, including procurement of raw materials; • Establishing sales, marketing and distribution capabilities and launching commercial sales of our products, if and when approved, whether alone or in combination with other products; • Sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials and commercialization activities; • Effectively competing with other therapies; • Developing and implementing successful marketing and reimbursement strategies; 54 • Obtaining and maintaining patent, trade secret and other intellectual property protection and regulatory exclusivity for our product candidates; and • Maintaining a continued acceptable safety profile of any product following approval, if any.
Because of the clinical stage of development of our product candidates, our ability to eventually generate significant revenues from product sales will depend on a number of factors, including: • Identification of additional target antigens for desired indications; • Identification and development of D-Domain-based binding regions that bind to the desired target antigens; • Successful completion of preclinical studies; • Submission of INDs or other regulatory applications for our planned clinical trials or future clinical trials and authorizations from regulators to initiate clinical trials; 58 • Successful enrollment in, and completion of, clinical trials; • Achieving favorable results from clinical trials; • Receipt of marketing approvals from applicable regulatory authorities; • Establishing and maintaining sufficient manufacturing capabilities, whether internally or with third parties, for clinical and commercial supply, including procurement of raw materials; • Establishing sales, marketing and distribution capabilities and launching commercial sales of our products, if and when approved, whether alone or in combination with other products; • Sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials and commercialization activities; • Effectively competing with other therapies; • Developing and implementing successful marketing and reimbursement strategies; • Obtaining and maintaining patent, trade secret and other intellectual property protection and regulatory exclusivity for our product candidates; and • Maintaining a continued acceptable safety profile of any product following approval, if any.
Some of these provisions include: • A board of directors divided into three classes serving staggered three-year terms, such that not all members of the board will be elected at one time, which could delay the ability of stockholders to change the membership of a majority of our board of directors; • The exclusive right of our board of directors to elect a director to fill a vacancy created by the expansion of our board of directors or the resignation, death, disqualification or removal of a director, which prevents stockholders from being able to fill vacancies on our board of directors; • A prohibition on stockholder action through written consent, which requires that all stockholder actions be taken at an annual or special meeting of our stockholders; 106 • A requirement that special meetings of stockholders be called only by the chairperson of our board of directors, our Chief Executive Officer, our President, or our board of directors acting pursuant to a resolution adopted by a majority of our board of directors, which could delay the ability of our stockholders to force consideration of a proposal or to take action, including the removal of directors; • Advance notice requirements for stockholder proposals and nominations for election to our board of directors, which may discourage or deter a potential acquirer from conducting a solicitation of proxies to elect the acquirer’s own slate of directors or otherwise attempting to obtain control of us; • A requirement that no member of our board of directors may be removed from office by our stockholders except for cause and, in addition to any other vote required by law, upon the approval of not less than a majority of the shares present in person or by proxy at the meeting and entitled to vote, which could delay the ability of stockholders to change the membership of our board of directors; • A requirement of approval of not less than two-thirds of all outstanding shares of our voting stock to amend any bylaws by stockholder action or to amend specific provisions of our certificate of incorporation, which may inhibit the ability of an acquirer to affect such amendments to facilitate an unsolicited takeover attempt; and • The authority of the board of directors to issue preferred stock on terms determined by the board of directors without stockholder approval (except as required by Nasdaq rules), which preferred stock may include rights superior to the rights of the holders of common stock and could be used to significantly dilute the ownership of a hostile acquirer.
Some of these provisions include: • A board of directors divided into three classes serving staggered three-year terms, such that not all members of the board will be elected at one time, which could delay the ability of stockholders to change the membership of a majority of our board of directors; • The exclusive right of our board of directors to appoint a director to fill a vacancy created by the expansion of our board of directors or the resignation, death, disqualification or removal of a director, which prevents stockholders from being able to fill vacancies on our board of directors; • A prohibition on stockholder action through written consent, which requires that all stockholder actions be taken at an annual or special meeting of our stockholders; • A requirement that special meetings of stockholders be called only by the chairperson of our board of directors, our Chief Executive Officer, our President, or our board of directors acting pursuant to a resolution adopted by a majority of our board of directors, which could delay the ability of our stockholders to force consideration of a proposal or to take action, including the removal of directors; 108 • Advance notice requirements for stockholder proposals and nominations for election to our board of directors, which may discourage or deter a potential acquirer from conducting a solicitation of proxies to elect the acquirer’s own slate of directors or otherwise attempting to obtain control of us; • A requirement that no member of our board of directors may be removed from office by our stockholders except for cause and, in addition to any other vote required by law, upon the approval of not less than a majority of the shares present in person or by proxy at the meeting and entitled to vote, which could delay the ability of stockholders to change the membership of our board of directors; • A requirement of approval of not less than two-thirds of all outstanding shares of our voting stock to amend any bylaws by stockholder action or to amend specific provisions of our certificate of incorporation, which may inhibit the ability of an acquirer to affect such amendments to facilitate an unsolicited takeover attempt; and • The authority of the board of directors to issue preferred stock on terms determined by the board of directors without stockholder approval (except as required by Nasdaq rules), which preferred stock may include rights superior to the rights of the holders of common stock and could be used to significantly dilute the ownership of a hostile acquirer.
Our business could be harmed if those third parties fail to provide us with sufficient quantities of product supplies or product candidates or fail to do so at acceptable quality levels or prices or if we terminate our relationship for any reason including due to a change in ownership, operating strategy or financial standing. • We depend on Kite for certain development, manufacturing and commercialization activities with respect to certain of our product candidates pursuant to our collaboration with Kite.
Our business could be harmed if those third parties fail to provide us with sufficient quantities of product supplies or product candidates or fail to do so at acceptable quality levels or prices or if we terminate our relationship for any reason including due to a change in ownership, operating strategy or financial standing. 54 • We depend on Kite for certain development, manufacturing and commercialization activities with respect to certain of our product candidates pursuant to our collaboration with Kite.
Applications for our product candidates could fail to receive regulatory approval for many reasons, including but not limited to the following: 91 • The FDA or foreign health authorities may disagree with the design, implementation or data analyses of our clinical trials; • The FDA or foreign health authorities may determine that our product candidate(s) do not have adequate risk-benefit ratio or have undesirable or unintended side effects, toxicities or other characteristics that preclude our obtaining marketing approval or prevent or limit commercial use; • The population studied in the clinical program may not be sufficiently broad or representative to assure efficacy and safety in the full population for which we seek approval; • The FDA or foreign health authorities may disagree with our interpretation of data from preclinical studies or clinical trials; • The data collected from clinical trials of our product candidates may not be sufficient to support the submission of a BLA or other submission or to obtain regulatory approval in the United States or elsewhere; • The FDA or foreign health authorities may fail to approve the manufacturing processes, test procedures and specifications or facilities of third-party manufacturers with which we contract for clinical and commercial supplies; and • The approval policies or regulations of the FDA or foreign health authorities may significantly change in a manner rendering our clinical data insufficient for approval.
Applications for our product candidates could fail to receive regulatory approval for many reasons, including but not limited to the following: • The FDA or foreign health authorities may disagree with the design, implementation or data analyses of our clinical trials; 78 • The FDA or foreign health authorities may determine that our product candidate(s) do not have adequate risk-benefit ratio or have undesirable or unintended side effects, toxicities or other characteristics that preclude our obtaining marketing approval or prevent or limit commercial use; • The population studied in the clinical program may not be sufficiently broad or representative to assure efficacy and safety in the full population for which we seek approval; • The FDA or foreign health authorities may disagree with our interpretation of data from preclinical studies or clinical trials; • The data collected from clinical trials of our product candidates may not be sufficient to support the submission of a BLA or other submission or to obtain regulatory approval in the United States or elsewhere; • The FDA or foreign health authorities may fail to approve the manufacturing processes, test procedures and specifications or facilities of third-party manufacturers with which we contract for clinical and commercial supplies; and • The approval policies or regulations of the FDA or foreign health authorities may significantly change in a manner rendering our clinical data insufficient for approval.
Depending on the facts and circumstances, we could be subject to criminal penalties if we knowingly obtain, use, or disclose individually identifiable health information maintained by a HIPAA-covered entity in a manner that is not authorized or permitted by HIPAA. 100 In addition, the California Consumer Privacy Act (CCPA) took effect in January 2020 and became enforceable in July 2020.
Depending on the facts and circumstances, we could be subject to criminal penalties if we knowingly obtain, use, or disclose individually identifiable health information maintained by a HIPAA-covered entity in a manner that is not authorized or permitted by HIPAA. In addition, the California Consumer Privacy Act (CCPA) took effect in January 2020 and became enforceable in July 2020.
Inadequate funding for the FDA, the SEC and other government agencies could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
Inadequate funding and other disruptions for the FDA, the SEC and other government agencies could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
CAR-T therapies for cancer indications, including anito-cel, are administered following a lymphodepletion regimen and often bridging therapies, these therapies are associated with risks of adverse events. As we expand clinical study of our T-cell based immunotherapies to other indications outside of cancer, such as autoimmune indications, we may use similar, intensive protocols to manage the risk of adverse events.
CAR-T therapies for cancer indications, including anito-cel, are administered following a lymphodepletion regimen and often bridging therapies, these therapies are associated with risks of adverse events. As we expand clinical study of our T-cell based immunotherapies to other indications outside of cancer, 66 such as autoimmune indications, we may use similar, intensive protocols to manage the risk of adverse events.
It is possible that a court could find these types of provisions to be inapplicable or unenforceable, and if a court were to find either exclusive-forum provision in our amended and restated bylaws to be inapplicable or unenforceable in an action, we may incur additional costs associated with resolving the dispute in other jurisdictions, which could seriously harm our business.
It is possible that a court could find these types of provisions to be inapplicable or unenforceable, and if a court were to find either 109 exclusive-forum provision in our amended and restated bylaws to be inapplicable or unenforceable in an action, we may incur additional costs associated with resolving the dispute in other jurisdictions, which could seriously harm our business.
In addition, if we are unable to effectively manage our outsourced activities or if the quality or accuracy of the services provided by consultants is compromised for any reason, our clinical trials may be extended, delayed or terminated, and we may not be able to obtain regulatory approval of our product candidates or otherwise advance our business.
In addition, if we are unable 85 to effectively manage our outsourced activities or if the quality or accuracy of the services provided by consultants is compromised for any reason, our clinical trials may be extended, delayed or terminated, and we may not be able to obtain regulatory approval of our product candidates or otherwise advance our business.
Our anticipated reliance on third-party manufacturers exposes us to the following risks: • We may be unable to identify manufacturers on acceptable terms or at all because the number of potential manufacturers is limited and the FDA must inspect any manufacturers for current cGMP. • Non-compliance of our third party manufacturers with requirements of our marketing application(s).
Our anticipated reliance on third-party manufacturers exposes us to the following risks: 93 • We may be unable to identify manufacturers on acceptable terms or at all because the number of potential manufacturers is limited and the FDA must inspect any manufacturers for current cGMP. • Non-compliance of our third-party manufacturers with requirements of our marketing application(s).
Any of the foregoing events could have a material adverse effect on our business, financial condition, results of operations, and prospects. 82 We may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent, which might adversely affect our ability to develop and market our products.
Any of the foregoing events could have a material adverse effect on our business, financial condition, results of operations, and prospects. We may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent, which might adversely affect our ability to develop and market our products.
There is no assurance that the FDA will not issue another clinical hold in the future. Any inability to successfully complete our clinical trials could result in additional costs to us or impair our ability to raise capital, generate revenues from product sales and enter into or maintain collaboration arrangements.
There is no assurance that the FDA will not issue another clinical hold in the future. 63 Any inability to successfully complete our clinical trials could result in additional costs to us or impair our ability to raise capital, generate revenues from product sales and enter into or maintain collaboration arrangements.
This is a class-wide boxed warning required for all approved CAR-T therapies. According to a November 2024 article, in view of reports that indicate the rate of secondary T-cell malignancies may be lower than initially thought, the FDA reported that the agency is reconsidering the need for such labeling.
This was a class-wide boxed warning required for all approved CAR-T therapies. According to a November 2024 article, in view of reports that indicate the rate of secondary T-cell malignancies may be lower than initially thought, the FDA reported that the agency is reconsidering the need for such labeling.
We seek to protect these trade secrets, in part, by entering into non-disclosure and confidentiality agreements with parties who have access to them, such as our employees, corporate collaborators, outside scientific collaborators, contract manufacturers, consultants, advisors and other third parties. We also enter into confidentiality and invention or patent assignment agreements with our employees and consultants.
We seek to protect these trade secrets, 104 in part, by entering into non-disclosure and confidentiality agreements with parties who have access to them, such as our employees, corporate collaborators, outside scientific collaborators, contract manufacturers, consultants, advisors and other third parties. We also enter into confidentiality and invention or patent assignment agreements with our employees and consultants.
Due to the high costs associated with cell therapies, patients are unlikely to use our product candidates unless coverage is provided or reimbursement is adequate to cover a significant portion of the cost of our product candidates. In the United States, no uniform policy of coverage and reimbursement for products exists among third party payors.
Due to the high 71 costs associated with cell therapies, patients are unlikely to use our product candidates unless coverage is provided or reimbursement is adequate to cover a significant portion of the cost of our product candidates. In the United States, no uniform policy of coverage and reimbursement for products exists among third party payors.
There can be no assurance that the ARC-T-cells, the SparX proteins, the TAG, anti-TAG and other parts of ACLX-001 and ACLX-002 will not trigger an adverse response, cause unintended off-target recognition, limit the expected activity of the product candidates or result in other negative outcomes.
There can be no assurance that the ARC-T-cells, the SparX proteins, the TAG, anti-TAG and other parts of ACLX-001, ACLX-002 and ACLX-004 will not trigger an adverse response, cause unintended off-target recognition, limit the expected activity of the product candidates or result in other negative outcomes.
We will conduct the iMMagine-1 trial for anito-cel and Kite will conduct all other development of the other Co-Promote Products. Kite will be responsible for commercialization of anito-cel and such other MM or lymphoma products, outside the United States, to the extent they are approved by the applicable regulatory authorities. Kite is responsible for manufacturing anito-cel under the collaboration.
We will conduct the iMMagine-1 trial for anito-cel and Kite will conduct all other development of the other Co-Promote Products. Kite will be responsible for commercialization of 95 anito-cel and such other MM or lymphoma products, outside the United States, to the extent they are approved by the applicable regulatory authorities. Kite is responsible for manufacturing anito-cel under the collaboration.
Drugs designated as breakthrough therapies by the FDA are also eligible for priority review if supported by clinical data at the time of the submission of the BLA. 93 Although Breakthrough Designation or access to any other expedited program may expedite the development or approval process, it does not change the standards for approval.
Drugs designated as breakthrough therapies by the FDA are also eligible for priority review if supported by clinical data at the time of the submission of the BLA. Although Breakthrough Designation or access to any other expedited program may expedite the development or approval process, it does not change the standards for approval.
Risks Related to Our Limited Operating History, Financial Condition, and Capital Requirements We have a limited operating history and have incurred significant losses since our inception, and we anticipate that we will continue to incur losses for the foreseeable future, which makes it difficult to assess our future viability. We are a clinical-stage biopharmaceutical company with a limited operating history.
Risks Related to Our Limited Operating History, Financial Condition, and Capital Requirements We have a limited operating history and have incurred significant losses since our inception, and we anticipate that we will continue to incur losses for the foreseeable future, which makes it difficult to assess our future viability. 56 We are a clinical-stage biopharmaceutical company with a limited operating history.
In the event of litigation or administrative proceedings, we cannot be certain that the claims in any of our issued patents will be considered valid by courts or administrative tribunals in the United States or foreign countries. 83 The strength of patents in the biotechnology and cell therapy fields involve complex legal and scientific questions and can be uncertain.
In the event of litigation or administrative proceedings, we cannot be certain that the claims in any of our issued patents will be considered valid by courts or administrative tribunals in the United States or foreign countries. The strength of patents in the biotechnology and cell therapy fields involve complex legal and scientific questions and can be uncertain.
The following examples are illustrative: • Pending patent applications that we own or may license may not lead to issued patents; • Patents, should they issue, that we own or may license, may not provide us with any competitive advantages, or may be challenged and held invalid or unenforceable; • Others may be able to develop and/or practice technology that is similar to our technology or aspects of our technology but that is not covered by the claims of any patents that we own or may license, should any such patents issue; • Third parties may compete with us in jurisdictions where we do not pursue and obtain patent protection; • We (or any licensors) might not have been the first to make the inventions covered by a pending patent application that we own or may license; • We (or any licensors) might not have been the first to file patent applications covering a particular invention; • Others may independently develop similar or alternative technologies without infringing our intellectual property rights; • We may not be able to obtain necessary licenses on reasonable terms or at all; 84 • Third parties may assert an ownership interest in our intellectual property and, if successful, such disputes may preclude us from exercising exclusive rights, or any rights at all, over that intellectual property; • We may need to initiate litigation or administrative proceedings to enforce and/or defend our patent rights, which will be costly whether we win or lose; • We may not be able to maintain the confidentiality of our trade secrets or other proprietary information; • We may not develop or in-license additional proprietary technologies that are patentable; and • The patents of others may have an adverse effect on our business.
The following examples are illustrative: • Pending patent applications that we own or may license may not lead to issued patents; • Patents, should they issue, that we own or may license, may not provide us with any competitive advantages, or may be challenged and held invalid or unenforceable; • Others may be able to develop and/or practice technology that is similar to our technology or aspects of our technology that is not covered by the claims of any patents that we own or may license; 100 • Third parties may compete with us in jurisdictions where we do not pursue and obtain patent protection; • We (or any licensors) might not have been the first to make the inventions covered by a pending patent application that we own or may license; • We (or any licensors) might not have been the first to file patent applications covering a particular invention; • Others may independently develop similar or alternative technologies without infringing our intellectual property rights; • We may not be able to obtain necessary licenses on reasonable terms or at all; • Third parties may assert an ownership interest in our intellectual property and, if successful, such disputes may preclude us from exercising exclusive rights, or any rights at all, over that intellectual property; • We may need to initiate litigation or administrative proceedings to enforce and/or defend our patent rights, which will be costly whether we win or lose; • We may not be able to maintain the confidentiality of our trade secrets or other proprietary information; • We may not develop or in-license additional proprietary technologies that are patentable; and • The patents of others may have an adverse effect on our business.
We cannot predict whether any such license would be available at all or whether it would be available on commercially reasonable terms. Furthermore, even in the absence of litigation, we may need or may choose to obtain licenses from third parties to advance our research or allow commercialization of our product candidates.
We cannot predict whether any such license would be available at all 101 or whether it would be available on commercially reasonable terms. Furthermore, even in the absence of litigation, we may need or may choose to obtain licenses from third parties to advance our research or allow commercialization of our product candidates.
The denial or delay of any such approval would delay commercialization and have a material adverse effect on our potential to generate revenue, our business and our results of operations. 51 • We will face increasing regulation as we advance our product candidates through clinical trials and pursue commercialization, if approved.
The denial or delay of any such approval would delay commercialization and have a material adverse effect on our potential to generate revenue, our business and our results of operations. • We will face increasing regulation as we advance our product candidates through clinical trials and pursue commercialization, if approved.
To the extent that the results of the trials are not satisfactory to the FDA or foreign health authorities for support of a marketing application, approval of our product candidates may be significantly delayed, or we may be required to expend significant additional resources, which may not be available to us, to conduct additional trials in support of potential approval of our product candidates.
To the extent that the results of the trials 62 are not satisfactory to the FDA or foreign health authorities for support of a marketing application, approval of our product candidates may be significantly delayed, or we may be required to expend significant additional resources, which may not be available to us, to conduct additional trials in support of potential approval of our product candidates.
These factors could include, among others, events such as liquidity constraints or failures, the ability to perform obligations under various types of financial, credit or liquidity agreements or arrangements, disruptions or instability in the financial services industry or financial markets, or concerns or negative expectations about the prospects for companies in the financial services industry.
These factors could include, among others, events such as liquidity constraints or failures, the ability to perform obligations under various types of financial, credit or liquidity agreements or arrangements, disruptions or instability in the financial services industry or financial markets, or concerns or negative expectations about the prospects for companies in the 84 financial services industry.
These measures could reduce the demand for our products, if approved, or impose additional pricing pressures on how much we can charge for our products if approved. 99 Legislative and regulatory proposals have been made to expand post-approval requirements and restrict sales and promotional activities for pharmaceutical products.
These measures could reduce the demand for our products, if approved, or impose additional pricing pressures on how much we can charge for our products if approved. Legislative and regulatory proposals have been made to expand post-approval requirements and restrict sales and promotional activities for pharmaceutical products.
Claims that we have violated individuals’ privacy rights, failed to comply with data protection laws, or breached our contractual obligations, even if we are not found liable, could be expensive and time consuming to defend and could result in adverse publicity that could harm our business.
Claims that we have violated individuals’ privacy rights, failed to comply with laws relating to privacy or data protection, or breached our contractual obligations, even if we are not found liable, could be expensive and time consuming to defend and could result in adverse publicity that could harm our business.
Later discovery of previously unknown problems with our product candidates, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things: • Restrictions on the marketing or manufacturing of our product candidates, withdrawal of the product from the market or voluntary or mandatory product recalls; • Fines, warning letters or holds on clinical trials; • Refusal by the FDA to approve pending applications or supplements to approved applications filed by us or suspension or revocation of license approvals; • Withdrawal of the drug from the market or voluntary or mandatory product recalls; • Adverse publicity, fines, warning letters or holds on clinical trials; 94 • Product seizure or detention, or refusal to permit the import or export of our product candidates; and • Injunctions or the imposition of civil or criminal penalties.
Later discovery of previously unknown problems with our product candidates, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things: • Restrictions on the marketing or manufacturing of our product candidates, withdrawal of the product from the market or voluntary or mandatory product recalls; • Fines, warning letters or holds on clinical trials; • Refusal by the FDA to approve pending applications or supplements to approved applications filed by us or suspension or revocation of license approvals; • Withdrawal of the drug from the market or voluntary or mandatory product recalls; • Adverse publicity, fines, warning letters or holds on clinical trials; 81 • Product seizure or detention, or refusal to permit the import or export of our product candidates; and • Injunctions or the imposition of civil or criminal penalties.
In the United States, numerous federal and state laws and regulations, including federal and state health information privacy laws, state data breach notification laws, and federal and state consumer protection laws, such as Section 5 of the Federal Trade Commission Act, govern the collection, use, disclosure and protection of health information and other personal information could apply to our operations.
In the United States, numerous federal and state laws and regulations, including federal and state health information privacy laws, state data breach notification laws, and federal and state consumer protection laws, such as Section 5 of the Federal Trade Commission Act, govern the collection, use, disclosure, transfer and protection of health information and other personal information and could apply to our operations.
Any national marketing authorization granted by a European Union Member State’s national authority can be used to support an application for its mutual recognition by other Member States. Marketing authorization applications can also be submitted directly to the Member State’s national competent authority under the national route (if the centralized route is not compulsory).
Any national marketing authorization granted by a European Union Member State’s national authority can be used to support an application for its mutual 75 recognition by other Member States. Marketing authorization applications can also be submitted directly to the Member State’s national competent authority under the national route (if the centralized route is not compulsory).
Pursuant to the Amended Standstill Agreement, Gilead has certain demand registration rights to require us to register the shares acquired by Gilead pursuant to the Purchase Agreement and second common stock purchase agreement with Gilead for resale. Once we register the shares held by Gilead, however, they can be freely sold in the public market.
Pursuant to the Amended Standstill Agreement, Gilead also has certain demand registration rights to require us to register the shares acquired by Gilead pursuant to the Purchase Agreement and second common stock purchase agreement with Gilead for resale. Once we register the shares held by Gilead, however, they can be freely sold in the public market.
Although we aligned with the FDA on modifications to the trial protocol and retrained our clinical trial sites in iMMagine-1 to enhance protocol adherence, there is no assurance that a site or another third party will not deviate from the trial protocol in the future.
Although we aligned with the FDA on modifications to the trial protocol and retrained our clinical trial sites in iMMagine-1 to enhance protocol 65 adherence, there is no assurance that a site or another third party will not deviate from the trial protocol in the future.
Defects of form in the preparation or filing of our patents or patent applications may exist, or may arise in the future, for example with respect to proper priority claims, inventorship, etc., although we are unaware of any such defects that we believe are of material import.
Defects of form in 97 the preparation or filing of our patents or patent applications may exist, or may arise in the future, for example with respect to proper priority claims, inventorship, etc., although we are unaware of any such defects that we believe are of material import.
For drugs that have been designated as breakthrough therapies, interaction and communication between the FDA and the sponsor of the trial can help to identify the most efficient path for clinical development while minimizing the number of patients placed in ineffective control regimens.
For drugs that have been designated as breakthrough therapies, interaction and communication between the FDA and the sponsor of the trial can help to identify the most efficient path for clinical development while minimizing the number 80 of patients placed in ineffective control regimens.
Clinical trials conducted in one country may not be accepted by regulatory authorities in other countries, and regulatory approval in one country does not mean that regulatory approval will be obtained in any other country. Approval procedures vary among countries and can involve additional product testing and validation and additional administrative review periods.
Clinical trials conducted in one country may not be accepted by regulatory authorities in other countries, and regulatory approval in one country does not mean that regulatory approval will be obtained in any other country. Approval procedures vary among countries and can involve additional product testing and validation 82 and additional administrative review periods.
Furthermore, our products may be subject to U.S. and foreign export controls, trade sanctions and import laws and regulations. Governmental regulation of the import or export of our products, or our failure to obtain any required import or export authorization for our products, when applicable, could harm our business.
Furthermore, our products may be subject to U.S. and foreign export controls, trade sanctions and import laws and regulations. Governmental regulation of the import or export of our products, or our failure to obtain any required import or export authorization 76 for our products, when applicable, could harm our business.
In addition to the factors discussed in this Risk Factors section, and elsewhere in this Annual Report on Form 10-K, these factors include: • Our decision to initiate a clinical trial, not to initiate a clinical trial, or to terminate an existing clinical trial; • The commencement, enrollment, or results of the clinical trials of our product candidates or any future clinical trials we may conduct, or changes in the development status of our product candidates; • Results from ongoing clinical trials and future clinical trials of our competitors; • Any delay in our regulatory filings for our product candidates and any adverse development or perceived adverse development with respect to the applicable regulatory authority’s review of such filings, including without limitation the FDA’s issuance of a “refusal to file” letter or a request for additional information; 103 • Our failure to achieve product development goals in the time frames we announce; • Adverse results or delays in clinical trials; • Adverse regulatory decisions, including failure to receive regulatory approval of our product candidates; • Changes in laws or regulations applicable to our product candidates, including, but not limited to, clinical trial requirements for approvals; • Adverse developments concerning our manufacturers; • Our inability to obtain adequate supply for any product candidate, or any component thereof, or approved product or inability to do so at acceptable prices; • Our inability to establish collaborations if needed; • Our failure to commercialize our product candidates; • Unanticipated serious safety concerns related to the use of our product candidates; • Introduction of new products or other therapies offered by us or our competitors; • Announcements of significant acquisitions, strategic partnerships, joint ventures, or capital commitments by us or our competitors; • Additions or departures of key scientific or management personnel; • Our ability to effectively manage our growth; • The size and growth of our initial cancer or autoimmune target markets and the markets of any other indications that we choose to target; • Our ability to successfully treat additional types of cancers or at different stages; • Our ability to successfully treat the indications outside of cancer, such as autoimmune indications, that we pursue; • Actual or anticipated variations in quarterly operating results; • Our cash position; • Our failure to meet the estimates and projections of the investment community or that we may otherwise provide to the public; • Publication of research reports about us or our industry, or positive or negative recommendations or withdrawal of research coverage by securities analysts; • Changes in the market valuations of similar companies; • Our operating performance and the performance of other similar companies; • Overall performance of the equity markets; • The expiration of contractual lock-up or standstill agreements; • Sales of our common stock by us or our stockholders in the future; • Trading volume of our common stock, which may be limited; 104 • Changes in accounting practices; • Ineffectiveness of our internal controls; • Disputes or other developments relating to proprietary rights, including patents, litigation matters, and our ability to obtain patent protection for our technologies; • Significant lawsuits, including patent or stockholder litigation; • General political and economic conditions, such as global pandemics or geopolitical uncertainty and instability, including ongoing geopolitical tensions related to the Russia and Ukraine conflict, resulting sanctions imposed by the United States and other countries, and retaliatory actions taken by Russia in response to such sanctions, and also those related to military conflicts in the Middle East; and • Other events or factors, many of which are beyond our control.
In addition to the factors discussed in this Risk Factors section, and elsewhere in this Annual Report on Form 10-K, these factors include, without limitation: • Our decision to initiate a clinical trial, not to initiate a clinical trial, or to terminate an existing clinical trial; • The commencement, enrollment, or results of the clinical trials of our product candidates or any future clinical trials we may conduct, or changes in the development status of our product candidates; • Results from ongoing clinical trials and future clinical trials of our competitors; • Any delay in our regulatory filings for our product candidates and any adverse development or perceived adverse development with respect to the applicable regulatory authority’s review of such filings, including without limitation the FDA’s issuance of a “refusal to file” letter or a request for additional information; • Our failure to achieve product development goals in the time frames we announce; • Adverse results or delays in clinical trials; • Adverse regulatory decisions, including failure to receive regulatory approval of our product candidates; • Changes in laws or regulations applicable to our product candidates, including, but not limited to, clinical trial requirements for approvals; • Adverse developments concerning our manufacturers; • Our inability to obtain adequate supply for any product candidate, or any component thereof, or approved product or inability to do so at acceptable prices; • Our inability to establish collaborations if needed; • Our failure to commercialize our product candidates; • Unanticipated serious safety concerns related to the use of our product candidates; • Introduction of new products or other therapies offered by us or our competitors; • Announcements of significant acquisitions, strategic partnerships, joint ventures, or capital commitments by us or our competitors; • Additions or departures of key scientific or management personnel; • Our ability to effectively manage our growth; • The size and growth of our initial cancer or autoimmune target markets and the markets of any other indications that we choose to target; • Our ability to successfully treat additional types of cancers or at different stages; • Our ability to successfully treat the indications outside of cancer, such as autoimmune indications, that we pursue; • Actual or anticipated variations in our operating results; • Our cash position; • Our failure to meet the estimates and projections of the investment community or that we may otherwise provide to the public; • Publication of research reports about us or our industry, or positive or negative recommendations or withdrawal of research coverage by securities analysts; • Changes in the market valuations of similar companies; • Our operating performance and the performance of other similar companies; • Overall performance of the equity markets; • The expiration of contractual lock-up or standstill agreements; • Sales of our common stock by us or our stockholders in the future; • Trading volume of our common stock, which may be limited; 106 • Changes in accounting practices; • Ineffectiveness of our internal controls; • Disputes or other developments relating to proprietary rights, including patents, litigation matters, and our ability to obtain patent protection for our technologies; • Significant lawsuits, including patent or stockholder litigation; • General political and economic conditions (including recent developments regarding global trade and inflation), such as global pandemics or geopolitical uncertainty and instability, including ongoing geopolitical tensions related to the Russia and Ukraine conflict, resulting sanctions imposed by the United States and other countries, and retaliatory actions taken by Russia in response to such sanctions, and also those related to military conflicts in the Middle East; and • Other events or factors, many of which are beyond our control.
Additionally, because all product candidates in our ARC-SparX platform use the ARC-T-cells, a failure with ACLX-001 or ACLX-002 will increase the actual or perceived likelihood that our other product candidates in the ARC-SparX platform will experience similar failures.
Additionally, because all product candidates in our ARC-SparX platform use the ARC-T-cells, a failure with ACLX-001 or ACLX-002 or ACLX-004 will increase the actual or perceived likelihood that our other product candidates in the ARC-SparX platform will experience similar failures.
We will also need to verify, such as through a manufacturing comparability study, that any new manufacturing process will produce our product candidate according to the specifications previously submitted to the FDA or another regulatory authority.
We will also need to verify, 94 such as through a manufacturing comparability study, that any new manufacturing process will produce our product candidate according to the specifications previously submitted to the FDA or another regulatory authority.
Moreover, clinical trial subjects about whom we or our potential collaborators obtain information, as well as the providers who share this information with us, may contractually limit our ability to use and disclose the information.
Moreover, clinical trial subjects about whom we or our potential collaborators obtain information, as well as the providers who share this information with us, may contractually limit our ability to use, transfer and disclose the information.
Risks Related to Our Business • We are highly dependent on our key personnel, and if we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy. • We expect to grow the size of our organization, and we may experience difficulties in managing this growth. • We may become exposed to costly and damaging product liability claims, either when testing our product candidates in the clinic or at the commercial stage, and our product liability insurance may not cover all damages from such claims. • Our business may be significantly adversely affected if events out of our control such as global pandemics, changes implemented by the new administration in the U.S., or geopolitical uncertainty and political instability disrupt our business, impact our people, or that of our third-party providers.
Risks Related to Our Business • We are highly dependent on our key personnel, and if we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy. • We expect to grow the size of our organization, and we may experience difficulties in managing this growth. • We may become exposed to costly and damaging product liability claims, either when testing our product candidates in the clinic or at the commercial stage, and our product liability insurance may not cover all damages from such claims. • Our business may be significantly adversely affected if events out of our control such as global pandemics, changes implemented by the new administration in the U.S., including changes in global trade policy, or geopolitical uncertainty and political instability disrupt our business, impact our people, or that of our third-party providers.
Changes in the leadership of the FDA and other federal agencies under the current presidential administration can result in changes in the funding, operations, and policies of the FDA and other federal agencies, which may impact our clinical development plans and timelines.
Changes in the leadership of the FDA and other federal agencies under the current administration can result in changes in the funding, operations, and policies of the FDA and other federal agencies, which may impact our clinical development plans and timelines.
While we are not aware of any downgrades, material losses, or other significant deterioration in the fair value of our cash equivalents and marketable securities since December 31, 2024, no assurance can be given that further deterioration of the global credit and financial markets would not negatively impact our current portfolio of cash equivalents and marketable securities or our ability to meet our financing objectives.
While we are not aware of any downgrades, material losses, or other significant deterioration in the fair value of our cash equivalents and marketable securities since December 31, 2025, no assurance can be given that further deterioration of the global credit and financial markets would not negatively impact our current portfolio of cash equivalents and marketable securities or our ability to meet our financing objectives.
Our amended and restated bylaws provide that the Court of Chancery of the State of Delaware (or, if the Court of Chancery does not have jurisdiction, another state court in Delaware) is the exclusive forum for the following (except for any claim as to which such court determines that there is an indispensable party not subject to the jurisdiction of such court (and the indispensable party does not consent to the personal jurisdiction of such court within 10 days following such determination), which is vested in the exclusive jurisdiction of a court or forum other than such court or for which such court does not have subject matter jurisdiction): • Any derivative action or proceeding brought on our behalf; • Any action asserting a claim of breach of fiduciary duty; • Any action asserting a claim against us arising under the Delaware General Corporation Law (DGCL), our amended and restated certificate of incorporation or our amended and restated bylaws; and • Any action asserting a claim against us that is governed by the internal-affairs doctrine.
Our amended and restated bylaws provide that the Court of Chancery of the State of Delaware (or, if the Court of Chancery does not have jurisdiction, another state court in Delaware) is the exclusive forum for the following (except for any claim as to which such court determines that there is an indispensable party not subject to the jurisdiction of such court (and the indispensable party does not consent to the personal jurisdiction of such court within 10 days following such determination), which is vested in the exclusive jurisdiction of a court or forum other than such court or for which such court does not have subject matter jurisdiction): • Any derivative action or proceeding brought on our behalf; • Any action asserting a claim of breach of fiduciary duty; • Any action asserting a claim against us arising under the DGCL, our amended and restated certificate of incorporation or our amended and restated bylaws; and • Any action asserting a claim against us that is governed by the internal-affairs doctrine.
Any delay in obtaining, or inability to obtain, applicable regulatory approval for any of our product candidates would delay or prevent commercialization of our product candidates and would materially adversely impact our business, results of operations and prospects. 90 The FDA regulatory approval process is lengthy, time-consuming and inherently unpredictable, and we may experience significant delays in the clinical development and regulatory approval of our product candidates or be unable to generate product revenue.
Any delay in obtaining, or inability to obtain, applicable regulatory approval for any of our product candidates would delay or prevent commercialization of our product candidates and would materially adversely impact our business, results of operations and prospects. 77 The FDA regulatory approval process is lengthy, time-consuming and inherently unpredictable, and we may experience significant delays in the clinical development and regulatory approval of our product candidates or be unable to generate product revenue.
Based on these and other factors, hospitals and payors may decide that the benefits of this new therapy do not or will not outweigh its costs. 56 Additionally, in developing our product candidates, we have not exhaustively explored different options in the design and method for manufacturing ddCARs, ARC-T-cells and SparX proteins.
Based on these and other factors, hospitals and payors may decide that the benefits of this new therapy do not or will not outweigh its costs. 60 Additionally, in developing our product candidates, we have not exhaustively explored different options in the design and method for manufacturing ddCARs, ARC-T-cells and SparX proteins.
Failure can occur at any time during clinical development, and, there is a high risk of failure and we may never succeed in developing marketable products. 57 The results of preclinical studies and earlier clinical trials of our product candidates may not be predictive of the results of later-stage clinical trials, particularly because earlier trials have smaller numbers of subjects tested.
Failure can occur at any time during clinical development, and there is a high risk of failure and we may never succeed in developing marketable products. 61 The results of preclinical studies and earlier clinical trials of our product candidates may not be predictive of the results of later-stage clinical trials, particularly because earlier trials have smaller numbers of subjects tested.
During the course of treatment, patients may suffer adverse events, including death, for reasons that may be related to our product candidates or course of treatment.
During the course of treatment, patients may suffer adverse events, including death, for reasons that may be related to our product candidates or course of 89 treatment.
We cannot assure you that, upon inspection, such regulatory authorities will determine that any of our clinical trials comply with the GCP regulations. In addition, our clinical trials must be conducted with biologic product produced under cGMP regulations and may require a significant number of test patients.
We cannot assure you that, upon inspection, such regulatory authorities will determine that any of our clinical trials comply with the GCP regulations. In addition, our clinical trials must be conducted with biologic products produced under cGMP regulations and may require a significant number of test patients.
If we face such litigation, it could result in substantial costs and a diversion of management’s attention and resources, which could harm our business, operating results, or financial condition. 108 Item 1B. Unreso lved Staff Comments. None.
If we face such litigation, it could result in substantial costs and a diversion of management’s attention and resources, which could harm our business, operating results, or financial condition. 110 Item 1B. Unreso lved Staff Comments. None.
In addition, we expect to also compete with companies developing: • T-cells with CARs that are reactive to tumor associated antigens; • T-cells with T-cell receptors (TCRs) that are reactive to tumor associated antigens; • T-cells with adapter platforms; • Bispecifics that bring T-cells and diseased cells into close proximity with each other; • Other immune cells that can be targeted using antibodies; 66 • Natural killer (NK)-based cell therapies; • In vivo CAR-T therapeutics; and • Allogeneic cell therapies.
In addition, we expect to also compete with companies developing: • T-cells with CARs that are reactive to tumor associated antigens; • T-cells with T-cell receptors (TCRs) that are reactive to tumor associated antigens; • T-cells with adapter platforms; • Bispecifics, trispecifics, and other multispecifics that bring T-cells and diseased cells into close proximity with each other; • Other immune cells that can be targeted using antibodies; • Natural killer (NK)-based cell therapies; • In vivo CAR-T therapeutics; and • Allogeneic cell therapies.
If we or any of our licensors is forced to grant a license to third parties with respect to any patents relevant to our business, our competitive position may be impaired, and our business, financial condition, results of operations, and prospects may be adversely affected.
If we or any of our licensors are forced to grant a license to third parties with respect to any patents relevant to our business, our competitive position may be impaired, and our business, financial condition, results of operations, and prospects may be adversely affected.
As a result of the 2022 ESPP, our stockholders may experience additional dilution. On January 1, 2025, the number of shares available for issuance under the 2022 ESPP was increased by 312,500 additional shares.
As a result of the 2022 ESPP, our stockholders may experience additional dilution. On January 1, 2026, the number of shares available for issuance under the 2022 ESPP was increased by 312,500 additional shares.
On January 1, 2022, a provision of the legislation commonly known as the Tax Cuts and Jobs Act of 2017 (the TCJA) went into effect, eliminating the option to deduct domestic research and development costs in the year incurred and instead requiring taxpayers to amortize such costs over five years.
On January 1, 2022, a provision of the legislation commonly known as the Tax Cuts and Jobs Act of 2017 (the TCJA) went into effect, eliminating the option to deduct domestic and foreign research and development costs in the year incurred and instead requiring taxpayers to amortize domestic research and development costs over five years and foreign research and development costs over fifteen years.
We currently have no sales organization and have a limited marketing organization and limited experience in marketing cell therapy products. If we are unable to establish adequate marketing and sales capabilities or establish or maintain relationships with third parties to market and sell our product candidates, if approved, we may not be able to generate product revenue.
If we are unable to establish adequate marketing and sales capabilities or establish or maintain relationships with third parties to market and sell our product candidates, if approved, we may not be able to generate product revenue. We currently have no sales or distribution capabilities and have limited marketing capabilities and limited experience in marketing cell therapy products.
While the extent of the impact of the COVID-19 pandemic on our business and financial results to date has been limited, the lasting effects of the pandemic on the drug development industry remains uncertain. Another prolonged public health crisis could have a material negative impact on our business, financial condition and operating results.
While the extent of the impact of the COVID-19 pandemic on our business and financial results to date was limited, the lasting effects of the pandemic on the drug development industry remains uncertain. Another prolonged public health crisis could have a material negative impact on our business, financial condition and operating results.
In addition, California has recently enacted a temporary suspension on the use of state net operating loss carryforwards for certain businesses, which may adversely affect our company if it earns taxable income in the impacted tax years. Other state tax limitations may apply.
In addition, California has recently enacted a temporary suspension on the use of state net operating loss carryforwards for certain businesses, which may adversely affect our company if it earns taxable income in the impacted tax years.
We have pending U.S. and foreign patent applications in our portfolio; however, we cannot predict: • If and when patents will issue based on our patent applications; • The scope of protection of any patent issuing based on our patent applications; • The degree and range of protection any issued patents will afford us against competitors including whether third parties will find ways to invalidate or otherwise circumvent our patents; • Whether any of our intellectual property will provide any competitive advantage; • Whether or not others will obtain patents claiming aspects similar to those covered by our patents and patent applications; • Whether we will need to initiate or defend litigation or administrative proceedings to enforce and/or defend our patent rights, which may be costly whether we win or lose; or • Whether the patent applications that we own or may in-license will result in issued patents with claims that cover our product candidates or uses thereof in the United States or in other foreign countries.
We have pending U.S. and foreign patent applications in our portfolio; however, we cannot predict: • If and when patents will issue based on our patent applications; • The scope of protection of any patent issuing based on our patent applications; • The degree and range of protection any issued patents will afford us against competitors including whether third parties will find ways to invalidate or otherwise circumvent our patents; • Whether any of our intellectual property will provide any competitive advantage; • Whether or not others will obtain patents claiming aspects similar to those covered by our patents and patent applications; • Whether we will need to initiate or defend litigation or administrative proceedings to enforce and/or defend our patent rights, which may be costly whether we win or lose; or • Whether the patent applications that we own or may in-license will result in issued patents with claims that cover our product candidates or uses thereof in the United States or in other foreign countries. 99 We cannot be certain that the claims in our pending patent applications directed to our product candidates and/or technologies will be considered patentable by the USPTO or by patent offices in foreign countries.
In addition, we may experience difficulty in identifying appropriate target binding domains. • Our ARC-SparX platform is highly dependent on the success of both ACLX-001 and ACLX-002. • Clinical development is a lengthy, expensive and uncertain process.
In addition, we may experience difficulty in identifying appropriate target binding domains. • Our ARC-SparX platform is highly dependent on the success of ACLX-001, ACLX-002 and ACLX-004. 53 • Clinical development is a lengthy, expensive and uncertain process.
Risks Related to Development of Our Product Candidates • Although the FDA has previously issued and lifted one partial clinical hold on anito-cel, there is no assurance that the safety measures included in our clinical protocols will be effective at mitigating the risk of future serious adverse events or that the FDA or DSMB will not issue another clinical hold in the future. • We have no products approved for commercial sale and have only recently begun clinical trials to test our first product candidates in humans, which may make it difficult for you to evaluate our current business and predict our future success and viability. • Our ddCAR and ARC-SparX platforms represent novel and unproven approaches to treatment, which makes it difficult to predict the timing, results and costs of product candidate development and the likelihood of obtaining regulatory approval.
Risks Related to Development of Our Product Candidates • Although the FDA has previously issued and lifted one partial clinical hold on anito-cel, there is no assurance that the safety measures included in our clinical protocols will be effective at mitigating the risk of future serious adverse events or that the FDA or DSMB will not issue another clinical hold in the future. • We have no products approved for commercial sale and have only one product candidate in late-stage clinical development, which may make it difficult for you to evaluate our current business and predict our future success and viability. • Our ddCAR and ARC-SparX platforms represent novel and unproven approaches to treatment, which makes it difficult to predict the timing, results and costs of product candidate development and the likelihood of obtaining regulatory approval.
ACLX-001 is an immunotherapeutic combination composed of ARC-T-cells and bi-valent SparX proteins targeting BCMA, or SPRX001, for the treatment of rrMM. ACLX-002 is an immunotherapeutic combination composed of ARC-T-cells and monovalent SparX proteins targeting CD123, or SPRX002, for the treatment of relapsed or refractory AML and high-risk MDS.
ACLX-001 is an immunotherapeutic combination composed of ARC-T-cells and SparX proteins targeting BCMA, or SPRX001, for the treatment of rrMM. ACLX-002 is an immunotherapeutic combination composed of ARC-T-cells and SparX proteins targeting CD123, or SPRX002, for the treatment of relapsed or refractory AML and high-risk MDS.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeFor additional information regarding whether any risks from cybersecurity threats, including as a result of any previous cybersecurity incidents, have materially affected or are reasonably likely to materially affect our company, including our business strategy, results of operations, or financial condition, please refer to Item 1A, “Risk Factors,” in this annual report on Form 10-K. 109 Governance One of the key functions of our board of directors is informed oversight of our risk management process, including risks from cybersecurity threats.
Biggest changeFor additional information regarding whether any risks from cybersecurity threats, including as a result of any previous cybersecurity incidents, have materially affected or are reasonably likely to materially affect our company, including our business strategy, results of operations, or financial condition, please refer to Item 1A, “Risk Factors,” in this Annual Report on Form 10-K. 111 Governance One of the key functions of our board of directors is informed oversight of our risk management process, including risks from cybersecurity threats.

Item 2. Properties

Properties — owned and leased real estate

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Removed
In October 2018, we entered into an operating lease agreement for 22,930 square feet of office and laboratory space in Gaithersburg, Maryland pursuant to a lease agreement that expires on January 31, 2030.
Removed
On January 30, 2024, we entered into an Assignment of Lease with a third party sublessee, pursuant to which we agreed to transfer and assign to a sublessee all of our rights, title, and interest under the Gaithersburg, Maryland lease.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeItem 3. Legal Proceedings. From time to time, we may become involved in litigation or other legal proceedings. As of December 31, 2024 we were not a party to any litigation or legal proceedings that, in the opinion of our management, are likely to have a material adverse effect on our business.
Biggest changeItem 3. Legal Proceedings. From time to time, we may become involved in litigation or other legal proceedings. As of December 31, 2025, we were not a party to any litigation or legal proceedings that, in the opinion of our management, are likely to have a material adverse effect on our business.

Item 4. Mine Safety Disclosures

Mine Safety Disclosures — required of mining issuers

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Biggest changeWe expect our operating expenses and capital requirements will increase substantially in connection with our ongoing activities, as we: • Advance the clinical program for anito-cel and subsequent clinical trials focused on earlier lines of therapy in collaboration with our partner Kite; 113 • Grow our supply and contract manufacturing infrastructure to support the continued development of anito-cel and our other product candidates; • Initiate clinical trials to evaluate anito-cel in other indications outside of oncology, such as generalized myasthenia gravis; • Initiate or continue to advance clinical trials to evaluate our clinical-stage ARC-SparX product candidates, ACLX-001 and ACLX-002, and other preclinical pipeline programs; • Expand our pipeline of product candidates, including through our own product discovery and development efforts or through acquisition or in-licensing; • Continue to develop our proprietary platforms to extend their use; • Attract, hire, and retain additional clinical, scientific, manufacturing, management and administrative personnel; • Add operational, financial, and management information systems and personnel, including personnel to support our product development, as well as to support us as a public reporting company; • Determine and execute our long-term manufacturing strategy for anito-cel in collaboration with our partner Kite; • Pursue regulatory approval of product candidates that successfully complete clinical trials; • Establish a sales, marketing and distribution infrastructure to commercialize any product candidate for which we may obtain regulatory approval; and • Obtain, maintain, expand and protect our intellectual property portfolio.
Biggest changeWe expect our operating expenses and capital requirements will increase substantially in connection with our ongoing activities, as we: • Establish a sales, marketing and distribution infrastructure to commercialize any product candidate for which we may obtain regulatory approval; • Advance the clinical program for anito-cel and subsequent clinical trials focused on earlier lines of therapy in collaboration with our partner Kite; • Pursue regulatory approval of product candidates that successfully complete clinical trials; • Attract, hire, and retain additional clinical, scientific, manufacturing, management, administrative and commercial personnel; • Add operational, financial, and management information systems and personnel, including personnel to support our product development; • Determine and execute our long-term manufacturing strategy for anito-cel in collaboration with our partner Kite; • Grow our supply and contract manufacturing infrastructure to support the continued development of anito-cel and our other product candidates; • Initiate clinical trials to evaluate anito-cel in other indications outside of oncology; • Initiate or continue to advance clinical trials to evaluate our clinical-stage candidates, such as anito-cel in non-oncology indications, including selected autoimmune disorders; and ARC-SparX product candidates, such as ACLX-001, ACLX-002, and other preclinical pipeline programs such as ACLX-004; • Expand our pipeline of product candidates, including through our own product discovery and development efforts or through acquisition or in-licensing; • Continue to develop our proprietary platforms to expand their use; and • Obtain, maintain, expand and protect our intellectual property portfolio.
General and Administrative Expenses General and administrative expenses were $88.4 million for the year ended December 31, 2024 compared to $66.4 million for the year ended December 31, 2023, an increase of $22.1 million.
General and administrative expenses were $88.4 million for the year ended December 31, 2024 compared to $66.4 million for the year ended December 31, 2023, an increase of $22.1 million.
Other income, net Other income, net was $32.3 million for the year ended December 31, 2024 compared to $19.9 million for the year ended December 31, 2023, an increase of $12.4 million. This increase was driven primarily by higher marketable securities balances and a corresponding increase in the interest earned.
Other income, net was $32.3 million for the year ended December 31, 2024 compared to $19.9 million for the year ended December 31, 2023, an increase of $12.4 million. This increase was driven primarily by higher marketable securities balances and a corresponding increase in the interest earned.
Investing Activities Net cash used in investing activities of $183.0 million during the year ended December 31, 2024 consists of $597.3 million in purchases of marketable securities and $13.4 million in purchases of property and equipment, offset by $427.7 million in proceeds from maturities of marketable securities.
Net cash used in investing activities of $183.0 million during the year ended December 31, 2024 consists of $597.3 million in purchases of marketable securities and $13.4 million in purchases of property and equipment, offset by $427.7 million in proceeds from maturities of marketable securities.
Financing Activities Net cash used in financing activities of $24.1 million during the year ended December 31, 2024 consists of $39.8 million payments under our finance lease from our Lonza manufacturing services agreement which ended in December 2024, offset by $15.8 million in proceeds from the exercise of stock options and stock issued pursuant to the employee stock purchase plan.
Net cash used in financing activities of $24.1 million during the year ended December 31, 2024 consists of $39.8 million payments under our finance lease from our Lonza manufacturing services agreement which ended in December 2024, offset by $15.8 million in proceeds from the exercise of stock options and stock issued pursuant to the employee stock purchase plan.
Internal control over financial reporting includes those policies and procedures that pertain to the maintenance of records that in reasonable detail accurately and fairly reflect the transactions and dispositions of the assets of the issuer; provide reasonable assurance that transactions are recorded as necessary for to permit preparation of our consolidated financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the issuer are being made only in accordance with authorizations of management and directors of the issuer; and provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use or disposition of the issuer’s assets that could have a material effect on the financial statements.
Internal control over financial reporting includes those policies and procedures that pertain to the maintenance of records that in reasonable detail accurately and fairly reflect the transactions and dispositions of the assets of the issuer; provide reasonable assurance that transactions are recorded as necessary for to permit 127 preparation of our consolidated financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the issuer are being made only in accordance with authorizations of management and directors of the issuer; and provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use or disposition of the issuer’s assets that could have a material effect on the financial statements.
We will reconsider the use of the Black-Scholes model if additional information becomes available in the future that indicates another model would be more appropriate or if grants issued in future periods have characteristics that prevent their value from being reasonably estimated using this model. 121 The Black-Scholes option pricing model requires inputs based on certain subjective assumptions.
We will reconsider the use of the Black-Scholes model if additional information becomes available in the future that indicates another model would be more appropriate or if grants issued in future periods have characteristics that prevent their value from being reasonably estimated using this model. The Black-Scholes option pricing model requires inputs based on certain subjective assumptions.
The payment terms of these agreements may include nonrefundable upfront fees, cost-sharing arrangements, payments based upon the achievement of certain milestones, and royalty payments based on product sales derived from the collaboration. We exercise judgment in assessing those promised goods and services that are distinct and thus representative of performance obligations.
The payment terms of these agreements may include nonrefundable upfront fees, cost-sharing arrangements, payments based upon the achievement of certain milestones, and royalty payments based on product sales derived from the collaboration. 124 We exercise judgment in assessing those promised goods and services that are distinct and thus representative of performance obligations.
We do not expect to generate any meaningful revenue from product sales unless and until we obtain regulatory approval of, and commercialize any of, our product candidates, except that we recognize revenue under the Kite Collaboration Agreement and its amendment on a cost-to-cost percentage of completion basis applied to the total estimated transaction price.
We do not expect to generate any meaningful revenue from product sales unless and until we obtain both regulatory approval and commercialize any of, our product candidates, except that we recognize revenue under the Kite Collaboration Agreement and its amendment on a cost-to-cost percentage of completion basis applied to the total estimated transaction price.
We only apply the five-step model to contracts when it is probable that we will collect the consideration to which we are entitled in exchange for the goods or services we transfer to the customer. 120 We evaluate the promised goods or services in these agreements to determine which ones represent distinct performance obligations.
We only apply the five-step model to contracts when it is probable that we will collect the consideration to which we are entitled in exchange for the goods or services we transfer to the customer. We evaluate the promised goods or services in these agreements to determine which ones represent distinct performance obligations.
Our methodology for developing the assumptions used in the valuation model are as follows: Fair Value of Common Stock —See the subsection titled “Determination of the fair value of our common stock and fair value of total equity” below. Expected Dividend Yield —The expected dividend yield is based on our historical and expected dividend payouts.
Our methodology for developing the assumptions used in the valuation model are as follows: Fair Value of Common Stock —See the subsection titled “Determination of the fair value of our common stock and fair value of total equity” below. 125 Expected Dividend Yield —The expected dividend yield is based on our historical and expected dividend payouts.
The discount period is the period between the valuation date and the assumed change in control event date, with the assumption that all equity shares in the capital structure are paid out in cash. Expected Dividend Yield —The expected dividend yield is based on our historical and expected dividend payouts.
The discount period is the period between the valuation date and the assumed change in control event date, with the assumption that all equity shares in the capital structure are paid out in cash. 126 Expected Dividend Yield —The expected dividend yield is based on our historical and expected dividend payouts.
The increase in research and development expenses was primarily attributable to an increase in internal costs of $21.3 million due to higher personnel-related costs, of which $10.4 million was due to non-cash share-based compensation; an increase of $5.4 million in anito-cel programs; an increase of $8.6 million in costs relating to ACLX-002 program; and an increase of $17.7 million in costs relating to other pipeline programs (including ACLX-003 in AML, anito-cel in myasthenia gravis, and additional discovery programs).
The increase in research and development expenses was primarily attributable to an increase in internal costs of $21.3 million due to higher personnel-related costs, of which $10.4 million was due to non-cash share-based compensation; an increase of $5.4 million in anito-cel programs; an increase of $8.6 million in costs relating to ACLX-002 program; and an increase of $17.7 million in costs relating to other pipeline programs (including ACLX-004 in AML, anito-cel in myasthenia gravis, and additional discovery programs).
Other Income, net 116 Other income, net consists primarily of interest earned on our cash and cash equivalents, restricted cash, and marketable securities, net accretion and amortization on marketable securities and interest expense related to our finance lease obligations.
Other Income, net Other income, net consists primarily of interest earned on our cash and cash equivalents, restricted cash, and marketable securities, net accretion and amortization on marketable securities and interest expense related to our finance lease obligations.
Evaluation of Disclosure Controls and Procedures Under the supervision and with the participation of our management, including our principal executive officer and principal financial and accounting officer, we conducted an evaluation of the effectiveness of our disclosure controls and procedures as of the end of the fiscal year on December 31, 2024, as such term is defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act.
Evaluation of Disclosure Controls and Procedures Under the supervision and with the participation of our management, including our principal executive officer and principal financial and accounting officer, we conducted an evaluation of the effectiveness of our disclosure controls and procedures as of the end of the fiscal year on December 31, 2025, as such term is defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act.
Performance Graph The following graph shows the total stockholder’s return on an initial investment of $100 in cash at market close on February 4, 2022 (the first day of trading of our common stock), through December 31, 2024 for (i) our common stock, (ii) the Nasdaq Composite Index and (iii) the Nasdaq Biotechnology Index.
Performance Graph The following graph shows the total stockholder’s return on an initial investment of $100 in cash at market close on February 4, 2022 (the first day of trading of our common stock), through December 31, 2025 for (i) our common stock, (ii) the Nasdaq Composite Index and (iii) the Nasdaq Biotechnology Index.
Based on this evaluation, management concluded that our internal control over financial reporting was effective as of December 31, 2024. The effectiveness of our internal control over financial reporting has been audited by PricewaterhouseCoopers LLP, an independent registered public accounting firm, as stated in their report which appears herein.
Based on this evaluation, management concluded that our internal control over financial reporting was effective as of December 31, 2025. The effectiveness of our internal control over financial reporting has been audited by PricewaterhouseCoopers LLP, an independent registered public accounting firm, as stated in their report which appears herein.
Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Management conducted an evaluation of the effectiveness, as of December 31, 2024, of our internal control over financial reporting based on the framework in Internal Control-Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission in 2013.
Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Management conducted an evaluation of the effectiveness, as of December 31, 2025, of our internal control over financial reporting based on the framework in Internal Control-Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission in 2013.
As of December 31, 2024, we were unable to estimate the timing or likelihood of achieving the milestones or making future product sales. Our operating lease obligations primarily consist of lease payments on our research, lab and office facilities in Rockville, Maryland and Redwood City, California.
As of December 31, 2025, we were unable to estimate the timing or likelihood of achieving the milestones or making future product sales. Our operating lease obligations primarily consist of lease payments on our research, lab and office facilities in Rockville, Maryland and Redwood City, California.
Item 4. Mine Sa fety Disclosures. Not applicable. 110 PART II It em 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities. Our common stock trades under the symbol “ACLX” on the Nasdaq Global Select Market.
Item 4. Mine Sa fety Disclosures. Not applicable. 112 PART II It em 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities. Our common stock trades under the symbol “ACLX” on the Nasdaq Global Select Market.
Management’s Discussion and Analysis of Financial Condition and Results of Operations. You should read the following discussion and analysis of our financial condition and results of operations together with our consolidated financial statements and the related notes thereto included elsewhere in this Annual Report on Form 10-K for the year ended December 31, 2024.
Management’s Discussion and Analysis of Financial Condition and Results of Operations. You should read the following discussion and analysis of our financial condition and results of operations together with our consolidated financial statements and the related notes thereto included elsewhere in this Annual Report on Form 10-K for the year ended December 31, 2025.
Recent Sales of Unregistered Equity Securities There were no sales of unregistered securities by us during the year ended December 31, 2024 that were not previously reported in our quarterly reports on Form 10-Q and current reports on Form 8-K filed with the SEC.
Recent Sales of Unregistered Equity Securities There were no sales of unregistered securities by us during the year ended December 31, 2025 that were not previously reported in our quarterly reports on Form 10-Q and current reports on Form 8-K filed with the SEC.
A 10% change in the interest rates in effect on December 31, 2024 would not have a material effect on the fair market value of our cash equivalents and available-for-sale securities. It em 8. Financial Statements and Supplementary Data.
A 10% change in the interest rates in effect on December 31, 2025 would not have a material effect on the fair market value of our cash equivalents and available-for-sale securities. It em 8. Financial Statements and Supplementary Data.
Changes in Internal Control Over Financial Reporting There were no changes in our internal control over financial reporting that occurred during the most recently completed fiscal quarter that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting. 124 It em 9B. Other Information.
Changes in Internal Control Over Financial Reporting There were no changes in our internal control over financial reporting that occurred during the most recently completed fiscal quarter that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting. 128 It em 9B. Other Information.
We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future, and our net losses may fluctuate significantly from period to period, depending on the timing of and expenditures on our planned research and development activities.
We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future, and our net losses may fluctuate significantly from period to period, depending on the timing of and expenditures on our planned research and development activities and commercial readiness activities.
Based on the evaluation of our disclosure controls and procedures as of December 31, 2024, our principal executive officer and principal financial and accounting officer concluded that, as of such date, our disclosure controls and procedures were effective at a reasonable assurance level.
Based on the evaluation of our disclosure controls and procedures as of December 31, 2025, our principal executive officer and principal financial and accounting officer concluded that, as of such date, our disclosure controls and procedures were effective at a reasonable assurance level.
We believe we can address these limitations by engineering a new class of D-Domain powered cell therapies, including classical single infusion CAR-Ts called “ddCARs” and dosable and controllable universal CAR-Ts called “ARC-SparX”, to address hematologic cancers, solid tumors, and indications outside of oncology, such as autoimmune diseases.
We believe we can address these limitations by engineering a new class of D-Domain powered immunotherapies, including classical single infusion CAR-Ts called “ddCARs” and dosable and controllable universal CAR-Ts called “ARC-SparX”, to address hematologic cancers, solid tumors, and indications outside of oncology, such as autoimmune diseases.
Based on the available objective evidence during the year ended December 31, 2024, we believe it is not more likely than not that the tax benefits of our net deferred income tax assets may be realized. Accordingly, we did not record the tax benefits of net deferred income tax assets previously incurred as of December 31, 2024.
Based on the available objective evidence during the year ended December 31, 2025, we believe it is not more likely than not that the tax benefits of our net deferred income tax assets may be realized. Accordingly, we did not record the tax benefits of net 120 deferred income tax assets previously incurred as of December 31, 2025.
Not applicable. 125 PART III We will file a definitive Proxy Statement for our 2025 Annual Meeting of Stockholders, or the Proxy Statement, with the SEC, pursuant to Regulation 14A, not later than 120 days after the end of our fiscal year. Accordingly, certain information required by Part III has been omitted under General Instruction G(3) to Form 10-K.
Not applicable. 129 PART III We will file a definitive Proxy Statement for our 2026 Annual Meeting of Stockholders, or the Proxy Statement, with the SEC, pursuant to Regulation 14A, not later than 120 days after the end of our fiscal year. Accordingly, certain information required by Part III has been omitted under General Instruction G(3) to Form 10-K.
Holders of Our Common Stock As of February 21, 2025, there were approximately 16 holders of record of shares of our common stock. This number does not include stockholders for whom shares are held in “nominee” or “street” name.
Holders of Our Common Stock As of February 20, 2026, there were approximately 16 holders of record of shares of our common stock. This number does not include stockholders for whom shares are held in “nominee” or “street” name.
We believe cell therapies are one of the forward pillars of medicine, and our mission is to advance humanity by engineering cell therapies that are safer, more effective and more broadly accessible. Although cell therapies have shown benefits to date, cell therapies have primarily been constrained to existing biologic structures, which has limited their impact and opportunity.
We believe immunotherapies are one of the forward pillars of medicine, and our mission is to advance humanity by engineering immunotherapies that are safer, more effective and more broadly accessible. Although CAR-Ts have shown benefits to date, they have primarily been constrained to existing biologic structures, which has limited their impact and opportunity.
Research and development expenses account for a significant portion of our operating expenses and consist primarily of external and internal costs incurred in connection with our anito-cel program, the development of our ARC-SparX product candidates, and the ongoing discovery and development efforts for additional product candidates.
Research and development expenses account for a significant portion of our operating expenses and consist primarily of external and internal costs incurred in connection with our anito-cel program, the development of anito-cel for the treatment of certain non-oncology indications, ARC-SparX product candidates, and the ongoing discovery and development efforts for additional product candidates.
Liquidity and Capital Resources Since inception, we have incurred net losses and negative cash flows from operations and we expect to incur substantial additional losses in future periods. As of December 31, 2024, we had cash and cash equivalents and marketable securities of $625.7 million. To date, we have not generated any product revenue.
Liquidity and Capital Resources Since inception, we have incurred net losses and negative cash flows from operations and we expect to incur substantial additional losses in future periods. As of December 31, 2025, we had cash and cash equivalents and marketable securities of $520.1 million. To date, we have not generated any product revenue.
External expenses include: • Payments to third parties in connection with the clinical development of our product candidates, including contract research organizations (CROs) and consultants; • The cost of manufacturing products for use in our preclinical studies and clinical trials, including payments to contract manufacturing organizations (CMOs) and consultants; • Payments to third parties in connection with the preclinical development of our product candidates, including outsourced professional scientific development services, consulting research fees and for sponsored research arrangements with third parties; • Laboratory supplies used in the preclinical development of our product candidates; and • Allocated facilities, depreciation, and other expenses, which include direct or allocated expenses for IT, rent and maintenance of facilities.
External expenses include: • Payments to third parties in connection with the clinical development of our product candidates, including contract research organizations (CROs) and consultants; • The cost of manufacturing products for use in our preclinical studies and clinical trials, including payments to contract manufacturing organizations (CMOs) and consultants; • Payments to third parties in connection with the preclinical development of our product candidates, including outsourced professional scientific development services, consulting research fees and for sponsored research arrangements with third parties; and • Laboratory supplies used in the preclinical development of our product candidates.
This follows the completion of the technical transfer to Kite, which was announced in May 2024, as well as the transfer to Kite of the Investigational New Drug (IND) application for anito-cel in rrMM, which has been cleared by the U.S. Food and Drug Administration (FDA).
This follows the completion of the technical transfer to Kite, which was announced in May 2024, as well as the transfer of the Investigational New Drug (IND) application for anito-cel in MM, which has been cleared by the FDA.
Our ability to eventually generate significant revenues from product sales will depend on a number of factors, including: • Successful enrollment in, and completion of, clinical trials; • Sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials; • Achieving favorable results from clinical trials; • Receipt of marketing approvals from applicable regulatory authorities; • Establishing and maintaining sufficient manufacturing capabilities, whether internally or with third parties, including securing raw material supply; • Existence of, and our ability to identify, an addressable patient population for our product candidates; • Effectively competing with other therapies; • Maintaining a continued acceptable safety profile of any product following approval, if any; • Submission of INDs or other regulatory applications for our planned clinical trials or future clinical trials and authorizations from regulators to initiate clinical trials; • Identification of additional target antigens for desired indications; • Identification and engineering of D-Domain-based binding regions that bind to the desired target antigens; • Developing and implementing successful marketing and reimbursement strategies; • Obtaining and maintaining patent, trade secret, and other intellectual property protection and regulatory exclusivity for our product candidates; and • The market opportunities for certain of our product candidates may be limited to those patients who are ineligible for or have failed prior treatments and may be small, and our projections regarding the size of the addressable market may be incorrect.
Our ability to eventually generate significant revenues from product sales will depend on a number of factors, including: • Successful enrollment in, and completion of, clinical trials; 119 • Achieving favorable results from clinical trials; • Sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials; • Receipt of marketing approvals from applicable regulatory authorities; • Developing and executing successful sales, marketing and reimbursement strategies; • The accuracy of our projections regarding the total addressable market for certain of our product candidates, which may be incorrect due to a number of factors including, but not limited to, patient availability, competition, reimbursement coverage and access, system capacity or execution challenges; • Establishing and maintaining sufficient manufacturing capabilities, whether internally or with third parties, including securing raw material supply; • Existence of, and our ability to identify, an addressable patient population for our product candidates; • Effectively competing with other therapies; • Maintaining a continued acceptable safety profile of any product following approval, if any; • Submission of INDs or other regulatory applications for our planned clinical trials or future clinical trials and authorizations from regulators to initiate clinical trials; • Identification of additional target antigens for desired indications; • Identification and engineering of D-Domain-based binding regions that bind to the desired target antigens; and • Obtaining and maintaining patent, trade secret, and other intellectual property protection and regulatory exclusivity for our product candidates.
Our lead program is a BCMA-targeting ddCAR product candidate called “anito-cel”, which is currently being evaluated in our pivotal Phase 2 iMMagine-1 and the Phase 3 iMMagine-3 trials in patients with relapsed or refractory multiple myeloma (rrMM).
Our lead program is a BCMA-targeting ddCAR product candidate called “anito-cel”, which is currently being evaluated in our pivotal Phase 2 iMMagine-1, Phase 3 iMMagine-3, and Phase 2 GEM-AnitoFIRST trials in patients with multiple myeloma (MM).
We are exposed to market risk related to changes in interest rates. As of December 31, 2024 and 2023, we had cash, cash equivalents and marketable securities of $625.7 million and $729.2 million, respectively, primarily invested in U.S. government agency securities and treasuries, certificate of deposit, corporate bonds, commercial paper and money market accounts.
We are exposed to market risk related to changes in interest rates. As of December 31, 2025 and 2024, we had cash, cash equivalents and marketable securities of $520.1 million and $625.7 million, respectively, primarily invested in U.S. government agency securities and treasuries, certificate of deposit, and money market accounts.
Securities Trading Plans of Directors and Executive Officers During our last fiscal quarter, none of our directors or officers, as defined in Rule 16a-1(f), adopted or terminated a “Rule 10b5-1 trading arrangement” or a “non-Rule 10b5-1 trading arrangement,” as defined in Regulation S-K Item 408.
Securities Trading Plans of Directors and Executive Officers During our last fiscal quarter, none of our directors or officers, as defined in Rule 16a-1(f), adopted or terminated a “Rule 10b5-1 trading arrangement” or a “non-Rule 10b5-1 trading arrangement,” as defined in Regulation S-K Item 408. It em 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections.
Our historical results are not necessarily indicative of the results that may be expected for any period in the future. Overview We are a clinical-stage biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases.
Our historical results are not necessarily indicative of the results that may be expected for any period in the future. Overview We are a clinical-stage biotechnology company focused on delivering a new class of innovative immunotherapies for patients with cancer and other incurable diseases.
Income Tax Provision We have recorded an income tax expense of $2.1 million, $663 thousand and zero for the years ended December 31, 2024, 2023 and 2022, respectively.
Income Tax Provision We have recorded an income tax expense of $69.0 thousand, $2.1 million and $0.7 million for the years ended December 31, 2025, 2024 and 2023, respectively.
This graph shall not be deemed “soliciting material” or be deemed “filed” for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities under that section, and shall not be deemed to be incorporated by reference into any of our filings under the Securities Act, whether made before or after the date hereof and irrespective of any general incorporation language in any such filing. 111 $100 investment in stock or index Ticker 2/4/2022 12/31/2022 12/31/2023 12/31/2024 Arcellx ACLX $ 100 $ 75 $ 292 $ 404 NASDAQ Composite Index IXIC $ 100 $ 75 $ 108 $ 138 NASDAQ Biotech Index NBI $ 100 $ 104 $ 107 $ 106 It em 6. [Reserved.] 112 It em 7.
This graph shall not be deemed “soliciting material” or be deemed “filed” for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities under that section, and shall not be deemed to be incorporated by reference into any of our filings under the Securities Act, whether made before or after the date hereof and irrespective of any general incorporation language in any such filing. 113 $100 investment in stock or index Ticker 2/4/2022 12/31/2022 12/31/2023 12/31/2024 12/31/2025 Arcellx ACLX $ 100 $ 184 $ 330 $ 456 $ 388 NASDAQ Composite Index IXIC $ 100 $ 74 $ 106 $ 137 $ 165 NASDAQ Biotech Index NBI $ 100 $ 103 $ 106 $ 105 $ 139 It em 6. [Reserved.] 114 It em 7.
General and administrative expenses also include allocated facilities, depreciation, and other expenses, which include direct or allocated expenses for rent and maintenance of facilities and insurance, not otherwise included in research and development expenses, as well as professional fees for legal, patent, consulting, investor and public relations, accounting, and audit services.
General and administrative expenses also include facilities, depreciation, commercial readiness activities, and other expenses not otherwise included in research and development expenses, as well as professional fees for legal, patent, consulting, investor and public relations, accounting, and audit services.
The primary difference between the effective tax rate and the statutory tax rate relates to change in state deferred income tax rate.
The primary difference between the effective tax rate and the statutory tax rate relates to change in valuation allowance.
Cash Flows The following table sets forth a summary of the primary sources and uses of cash for each of the periods presented below (in thousands): Year Ended December 31, 2024 2023 2022 Net cash provided by (used in) operating activities $ (83,467 ) $ 207,573 $ (99,303 ) Net cash used in investing activities (183,045 ) (154,512 ) (117,674 ) Net cash provided by (used in) financing activities (24,087 ) 279,163 252,625 Net increase (decrease) in cash, cash equivalents, and restricted cash $ (290,599 ) $ 332,224 $ 35,648 Operating Activities Net cash used in operating activities during the year ended December 31, 2024 of $83.5 million was attributable to our net loss of $107.3 million, partially offset by adjustments to net loss of $54.7 million.
Cash Flows The following table sets forth a summary of the primary sources and uses of cash for each of the periods presented below (in thousands): Year Ended December 31, 2025 2024 2023 Net cash provided by (used in) operating activities $ (210,258 ) $ (83,467 ) $ 207,573 Net cash provided by (used in) investing activities 86,297 (183,045 ) (154,512 ) Net cash provided by (used in) financing activities 98,534 (24,087 ) 279,163 Net increase (decrease) in cash, cash equivalents, and restricted cash $ (25,427 ) $ (290,599 ) $ 332,224 Operating Activities Net cash used in operating activities during the year ended December 31, 2025 of $210.3 million was attributable to our net loss of $228.9 million, partially offset by adjustments to net loss of $76.2 million.
Existing cell therapy solutions, most of which use a biologic-based, single chain variable fragment (scFv) binding domain, tend to be difficult to manufacture, beneficial to a limited segment of patients, often result in high toxicity, and have narrow applicability in treatable indications.
Our novel synthetic binding scaffold, the D-Domain, is designed to overcome the limitations of traditional CAR-Ts. Existing CAR-T therapy solutions, most of which use a biologic-based, single chain variable fragment (scFv) binding domain, tend to be difficult to manufacture, beneficial to a limited segment of patients, often result in high toxicity, and have narrow applicability in treatable indications.
We are subject to all of the risks typically related to the development of new product candidates, and we may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may adversely affect our business.
We are subject to all of the risks typically related to the development of new product candidates, and we may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may adversely affect our business. Moreover, we expect to continue to incur additional costs associated with operating as a public company.
As a result, we expect that our research and development expenses will increase substantially in the foreseeable future as we continue to advance anito-cel, in multiple myeloma and other indications outside of oncology, through clinical development, the regulatory approval process and, if approved, commercial launch activities; initiate or continue to advance our ARC-SparX product candidates, including expanding ACLX-001 and ACLX-002; continue to discover and develop additional product candidates to expand our pipeline; maintain, expand, protect, and enforce our intellectual property portfolio; and hire additional personnel. 115 The successful development of our product candidates is highly uncertain, and we do not believe it is possible at this time to accurately project the nature, timing, and estimated costs of the efforts necessary to complete the development of, and obtain regulatory approval for, any of our product candidates.
As a result, we expect that our research and development expenses will increase substantially in the foreseeable future as we continue to advance anito-cel, in multiple myeloma and other indications outside of oncology, through clinical development, the regulatory approval process and, if approved, commercial launch activities; initiate or continue to advance non-oncology indications, including selected autoimmune disorders and our ARC-SparX product candidates, including expanding ACLX-001 and ACLX-002; continue to discover and develop additional product candidates to expand our pipeline; maintain, expand, protect, and enforce our intellectual property portfolio; and hire additional personnel.
Based on our expected operating cash requirements and capital expenditures, we believe our current cash and cash equivalents and investments in marketable securities are adequate to fund operations into 2027. 118 In May 2023, we entered into a sales agreement (Sales Agreement) with Stifel, Nicolaus & Company (Stifel) with respect to an at-the-market offering program under which we may issue and sell, from time to time and at our sole discretion, shares of our common stock, in an aggregate offering amount of up to $350.0 million.
In May 2023, we entered into a sales agreement (Sales Agreement) with Stifel, Nicolaus & Company (Stifel) with respect to an at-the-market offering program under which we may issue and sell, from time to time and at our sole discretion, shares of our common stock, in an aggregate offering amount of up to $350.0 million.
Disclosure controls and procedures are designed to ensure that information required to be disclosed by us in our Exchange Act reports is recorded, processed, summarized, and reported within the time periods specified in the SEC’s rules and forms, and that such information is accumulated and communicated to our management, including our principal executive officer and principal financial officer or persons performing similar functions, as appropriate to allow timely decisions regarding required disclosure. 123 Management recognizes that any controls and procedures, no matter how well designed and operated, can only provide reasonable assurance of achieving their objectives, and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures.
Disclosure controls and procedures are designed to ensure that information required to be disclosed by us in our Exchange Act reports is recorded, processed, summarized, and reported within the time periods specified in the SEC’s rules and forms, and that such information is accumulated and communicated to our management, including our principal executive officer and principal financial officer or persons performing similar functions, as appropriate to allow timely decisions regarding required disclosure.
Since our formation, we have devoted substantially all our resources to discovering and developing our product candidates. We have incurred significant operating losses to date. Our net losses were $107.3 million, $70.7 million, and $188.7 million for the years ended December 31, 2024, 2023 and 2022. Our accumulated deficit totaled $496.8 million as of December 31, 2024.
We have incurred significant operating losses to date. Our net losses were $228.9 million, $107.3 million, and $70.7 million for the years ended December 31, 2025, 2024 and 2023. Our accumulated deficit totaled $725.8 million as of December 31, 2025.
Recent Developments In December 2024, we announced preliminary data from our pivotal iMMagine-1 study in patients with rrMM, which were presented during an oral presentation at the 66th ASH Annual Meeting and Exposition.
In December 2025, we announced interim data from our pivotal iMMagine-1 study in patients with rrMM after three or more prior lines of therapy, which were presented during an oral presentation at the 67th ASH Annual Meeting and Exposition.
Results of Operations The following table summarizes our results of operations (in thousands, except percentages): Year Ended December 31, Change 2024 2023 2022 2024 vs 2023 2023 vs 2022 Collaboration revenue $ 107,936 $ 110,319 $ — $ (2,383 ) -2 % $ 110,319 100% Operating expenses: Research and development 157,093 133,849 149,555 23,244 17 % (15,706 ) -11 % General and administrative 88,414 66,350 41,704 22,064 33 % 24,646 59 % Total operating expenses 245,507 200,199 191,259 45,308 23 % 8,940 5 % Loss from operations (137,571 ) (89,880 ) (191,259 ) (47,691 ) 53 % 101,379 -53 % Interest and other income (expense), net 33,322 23,695 4,300 9,627 41 % 19,395 451 % Interest expense (1,030 ) (3,842 ) (1,720 ) 2,812 -73 % (2,122 ) 123 % Total other income, net 32,292 19,853 2,580 12,439 63 % 17,273 669 % Income tax expense (2,069 ) (663 ) — (1,406 ) 212 % (663 ) 100 % Net loss $ (107,348 ) $ (70,690 ) $ (188,679 ) $ (36,658 ) 52 % $ 117,989 -63 % Collaboration Revenue Collaboration revenue was $107.9 million for the year ended December 31, 2024 compared to $110.3 million for the year ended December 31, 2023.
Results of Operations The following table summarizes our results of operations (in thousands, except percentages): Year Ended December 31, Change 2025 2024 2023 2025 vs 2024 2024 vs 2023 Collaboration revenue $ 22,286 $ 107,936 $ 110,319 $ (85,650 ) -79 % $ (2,383 ) -2 % Operating expenses: Research and development 157,611 157,093 133,849 518 0 % 23,244 17 % General and administrative 117,758 88,414 66,350 29,344 33 % 22,064 33 % Total operating expenses 275,369 245,507 200,199 29,862 12 % 45,308 23 % Loss from operations (253,083 ) (137,571 ) (89,880 ) (115,512 ) 84 % (47,691 ) 53 % Interest and other income (expense), net 24,218 33,322 23,695 (9,104 ) -27 % 9,627 41 % Interest expense - (1,030 ) (3,842 ) 1,030 -100 % 2,812 -73 % Total other income, net 24,218 32,292 19,853 (8,074 ) -25 % 12,439 63 % Income tax expense (69 ) (2,069 ) (663 ) 2,000 -97 % (1,406 ) 212 % Net loss $ (228,934 ) $ (107,348 ) $ (70,690 ) $ (121,586 ) 113 % $ (36,658 ) 52 % Collaboration Revenue Collaboration revenue was $22.3 million for the year ended December 31, 2025 compared to $107.9 million for the year ended December 31, 2024, a decrease of $85.7 million.
This increase was driven primarily by an increase of $19.3 million in personnel related costs due to an increase in headcount ($16.2 million of which was due to non-cash stock-based compensation expense), $3.2 million in facilities costs, and $1.4 million in consulting fees.
This increase was driven primarily by an increase of $16.5 million in personnel-related costs, of which $7.9 million was due to non-cash stock-based compensation, an increase of $7.8 million in commercial readiness costs, and an increase of $1.3 million in facility costs.
We also are developing two clinical-stage ARC-SparX programs in Phase 1 trials, ACLX-001, which targets BCMA in rrMM, and our wholly-owned ACLX-002, which targets CD123 in relapsed or refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). In November 2023, Kite exercised its option under the Kite Collaboration Agreement to negotiate a license for ACLX-001.
We began dosing patients in a Phase 1 trial in generalized Myasthenia Gravis (gMG) in the second half of 2025.We also are advancing several ARC-SparX programs: ACLX-001, which targets BCMA in rrMM and for which Kite exercised its option under the Kite Collaboration Agreement to negotiate a license in November 2023; our wholly-owned ACLX-002, which targets CD123 in relapsed or refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS); and our wholly-owned ACLX-004, which targets CD33 and CD123 in relapsed or refractory AML. 115 Since our formation, we have devoted substantially all our resources to discovering and developing our product candidates.
Other income, net was $19.9 million for the year ended December 31, 2023 compared to $2.6 million for the year ended December 31, 2022, an increase of $17.3 million. This increase was driven primarily by higher overall cash and cash equivalents and marketable securities balances and a corresponding increase in the interest earned.
Other income, net Other income, net was $24.2 million for the year ended December 31, 2025 compared to $32.3 million for the year ended December 31, 2024, a decrease of $8.1 million. This decrease was driven primarily by lower marketable securities balances and a corresponding decrease in the interest earned.
Recently, Kite initiated a global Phase 3 randomized controlled clinical trial (iMMagine-3) of anito-cel in patients with second through fourth line rrMM. Kite will manufacture anito-cel for iMMagine-3.
In 2024, Kite initiated a global Phase 3 randomized controlled clinical trial (iMMagine-3) of anito-cel in patients with second through fourth line rrMM. Kite is manufacturing anito-cel for iMMagine-3 and expects the trial to be fully enrolled by mid-2026.
Application of the Monte Carlo simulation model required various subjective assumptions that represent management’s best estimates of the fair value of common stock, expected equity volatility, risk-free interest rate, discount period, expected dividend yield, and time to achievement of a performance condition: Fair Value of Common Stock and Fair Value of Total Equity —See the subsection titled “Determination of the fair value of our common stock and fair value of total equity” below. 122 Expected Equity Volatility —Due to the lack of a public market for our common stock (prior to our IPO) and the lack of company-specific historical and implied volatility data, we have based our computation of expected volatility on the historical volatility of a representative group of public companies with similar characteristics to us (e.g., public entities of similar size, complexity, stage of development, and industry focus).
Application of the Monte Carlo simulation model required various subjective assumptions that represent management’s best estimates of the fair value of common stock, expected equity volatility, risk-free interest rate, discount period, expected dividend yield, and time to achievement of a performance condition: Fair Value of Common Stock and Fair Value of Total Equity —See the subsection titled “Determination of the fair value of our common stock and fair value of total equity” below.
Research and Development Expenses The detail of our external and internal research and development costs is as follows (in thousands, except percentages): Year Ended December 31, Change 2024 2023 2022 2024 vs 2023 2023 vs 2022 External costs: anito-cel in rrMM $ 50,141 $ 73,530 $ 96,513 $ (23,389 ) -32 % $ (22,983 ) -24 % ACLX-001 1,975 2,939 8,764 (964 ) -33 % (5,825 ) -66 % ACLX-002 14,294 5,667 6,458 8,627 152 % (791 ) -12 % Other research and development costs 21,364 3,701 5,467 17,663 477 % (1,766 ) -32 % Total external costs 87,774 85,837 117,202 1,937 2 % (31,365 ) -27 % Internal costs 69,319 48,012 32,353 21,307 44 % 15,659 48 % Total research and development expenses $ 157,093 $ 133,849 $ 149,555 $ 23,244 17 % $ (15,706 ) -11 % 117 Research and development expenses were $157.1 million for the year ended December 31, 2024 compared to $133.8 million for the year ended December 31, 2023, an increase of $23.2 million.
Research and Development Expenses The detail of our external and internal research and development costs is as follows (in thousands, except percentages): Year Ended December 31, Change 2025 2024 2023 2025 vs 2024 2024 vs 2023 External costs: anito-cel in rrMM $ 32,719 $ 50,141 $ 73,530 $ (17,422 ) -35 % $ (23,389 ) -32 % ACLX-002 9,153 14,294 5,667 (5,141 ) -36 % 8,627 152 % Other research and development costs 27,132 23,339 6,640 3,793 16 % 16,699 251 % Total external costs 69,004 87,774 85,837 (18,770 ) -21 % 1,937 2 % Internal costs 88,607 69,319 48,012 19,288 28 % 21,307 44 % Total research and development expenses $ 157,611 $ 157,093 $ 133,849 $ 518 0 % $ 23,244 17 % Research and development expenses were $157.6 million for the year ended December 31, 2025 compared to $157.1 million for the year ended December 31, 2024, an increase of $0.5 million.
General and administrative expenses were $66.4 million for the year ended December 31, 2023 compared to $41.7 million for the year ended December 31, 2022, an increase of $24.6 million.
General and Administrative Expenses General and administrative expenses were $117.8 million for the year ended December 31, 2025 compared to $88.4 million for the year ended December 31, 2024, an increase of $29.3 million.
Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages, primarily due to the increased size and duration of later-stage clinical trials.
Internal expenses include employee-related costs, including salaries, related benefits, and share-based compensation expense for employees engaged in research and development functions, as well as facilities, depreciation and other expenses. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages, primarily due to the increased size and duration of later-stage clinical trials.
We cannot assure you that we will ever be profitable or generate positive cash flow from operating activities. Based on our expected operating cash requirements and capital expenditures, we believe our current cash and cash equivalents and investments in marketable securities are adequate to fund operations into 2027.
Adequate funding may not be available to us on acceptable terms or at all. Based on our expected operating cash requirements and capital expenditures, we believe our current cash and cash equivalents and investments in marketable securities are adequate to fund operations into 2028.
Net cash used in investing activities of $154.5 million during the year ended December 31, 2023 consists of $442.4 million in purchases of marketable securities and $21.4 million in purchases of property and equipment, offset by $309.3 million in proceeds from maturities of marketable securities. 119 Net cash used in investing activities of $117.7 million during the year ended December 31, 2022 consists of $273.7 million in purchases of marketable securities, offset by $158.3 million in proceeds from maturities of marketable securities and $2.3 million in purchases of lab equipment used in the development of our cell therapies.
Investing Activities Net cash provided by investing activities of $86.3 million during the year ended December 31, 2025 consists of $495.6 million in proceeds from maturities of marketable securities, offset by $407.0 million in purchases of marketable securities and $2.3 million in purchases of property and equipment.
Changes in operating assets and liabilities increased cash by $4.5 million, primarily due to increases of accounts payable and other current liabilities and accrued liabilities of $7.0 million, and increases in operating lease liabilities of $3.1 million, offset by decreases in prepaid assets and other current and non-current assets of $5.7 million.
Changes in operating assets and liabilities decreased cash by $57.5 million, primarily due to changes in contract liability to related party of $26.0 million, other non-current liabilities of $8.6 million, and accrued liabilities of $18.8 million.
Internal costs had an increase of $9.1 million in personnel related costs due to an increase in headcount ($4.0 million of which was due to non-cash stock-based compensation expense), an increase of $4.1 million in facilities costs, and an increase of $1.2 million in depreciation expenses.
The increase in research and development expenses was primarily attributable to an increase in internal costs of $19.3 million primarily due to $16.5 million increase in personnel-related costs, of which $8.9 million was due to non-cash share-based compensation, and $2.2 million increase in depreciation expense; and an increase of $3.8 million in costs relating to other pipeline programs (including anito-cel in myasthenia gravis and additional discovery programs).
We anticipate that our general and administrative expenses will increase as we increase our headcount to support the growth of the company.
We expect that our general and administrative expenses will increase as we expand our operating activities and increase our headcount to prepare for a potential commercial launch of anito-cel.
Outside of our collaboration with Kite, we intend to evaluate anito-cel for the treatment of certain non-oncology indications, including some autoimmune disorders. We received FDA clearance of an IND application and have initiated a Phase 1 trial in generalized myasthenia gravis (gMG) in 2024.
On February 22, 2026, we entered into an Agreement and Plan of Merger (the Merger Agreement) with Gilead and Purchaser, as described in more detail in "Pending Acquisition by Gilead" below (the Merger). Outside of our collaboration with Kite, we intend to evaluate anito-cel for the treatment of certain non-oncology indications, including some autoimmune disorders.
Net cash used in operating activities during the year ended December 31, 2022 of $99.3 million was primarily attributable to our net loss of $188.7 million, partially offset by adjustments to net loss of $84.9 million, primarily consisting of expensing of a right-of-use asset of $63.3 million, together with share-based compensation of $21.5 million, amortization of premiums and discounts on marketable securities of $2.1 million, and depreciation and amortization of property and equipment of $1.3 million.
Net cash used in operating activities during the year ended December 31, 2024 of $83.5 million was attributable to our net loss of $107.3 million, partially offset by adjustments to net loss of $54.7 million.
We have partnered anito-cel with Kite Pharma Inc., a Gilead company (Kite), through our co-development/co-commercialization collaboration agreement (as described in more detail in the section below titled “Components of Results of Operations - Revenue" included in this Annual Report on Form 10-K).
We have partnered anito-cel with Kite Pharma Inc., a Gilead company (Kite), through our co-development/co-commercialization collaboration agreement, as described in more detail in "Licenses and Collaboration" (Kite Collaboration Agreement). In 2024, we completed dosing in our pivotal Phase 2 clinical trial (iMMagine-1) of anito-cel in patients with fourth line or later relapsed or refractory MM (rrMM).
Collaboration revenue was $110.3 million for the year ended December 31, 2023 compared to zero for the year ended December 31, 2022, as we began recognizing collaboration revenue in 2023 under the Kite Collaboration Agreement and its amendment.
This decrease was primarily driven by completion of dosing and manufacturing of anito-cel in the iMMagine-1 trial in the fourth quarter of 2024. Collaboration revenue was $107.9 million for the year ended December 31, 2024 compared to $110.3 million for the year ended December 31, 2023.
Removed
Our novel synthetic binding scaffold, the D-Domain, is designed to overcome the limitations of traditional Chimeric Antigen Receptor T-cells (CAR-Ts).
Added
On December 23, 2025, we submitted a BLA for anito-cel to treat patients with fourth line or later rrMM to the FDA. On February 20, 2026, the FDA notified us that it has accepted our BLA with an anticipated PDUFA action date of December 23, 2026.
Removed
In November 2024, we announced with Kite that the first patients have been dosed in the global iMMagine-3 randomized control trial evaluating anito-cel in patients with rrMM exposed to an immunomodulatory (IMiD) drug and an anti-CD38 monoclonal antibody. 114 Components of Results of Operations Revenue We have not generated any revenue from product sales and do not expect to do so in the near future.
Added
We cannot assure you that we will ever be profitable or generate positive cash flow from operating activities.
Removed
Internal expenses include employee-related costs, including salaries, related benefits, and share-based compensation expense for employees engaged in research and development functions. Our Manufacturing Services Agreement with Lonza Houston, Inc. (Lonza) expired in December 2024.
Added
Based on our expected operating cash requirements and capital expenditures, we believe our current cash and cash equivalents and investments in marketable securities are adequate to fund operations into 2028. 116 Pending Acquisition by Gilead Merger Agreement On February 22, 2026, we entered into the Merger Agreement with Gilead and Purchaser.
Removed
Previously, we had identified embedded leases within the agreement, which had resulted in right-of-use assets that were expensed as research and development expenses in 2022 and 2023 as the right-of-use assets had no alternative future use.
Added
The Merger Agreement provides for the acquisition of the Company by Gilead in a two-step transaction, consisting of a tender offer followed by a subsequent merger of Purchaser with and into the Company (the Merger and together with the Offer and the other transactions contemplated by the Merger Agreement, the Transactions), with the Company continuing as the surviving corporation.
Removed
Research and development expenses were $133.8 million for the year ended December 31, 2023 compared to $149.6 million for the year ended December 31, 2022, a decrease of $15.7 million.
Added
Pursuant to the Merger Agreement, and upon the terms and subject to the conditions thereof, Purchaser will commence a tender offer (the Offer), to acquire all of our issued and outstanding shares of common stock, par value $0.001 per share (the Shares), other than any Shares owned immediately prior to the effective time of the Merger by the Company (including shares held in our treasury) and any Shares owned both as of the date of the commencement of the Offer and immediately prior to the effective time of the Merger by Gilead, Purchaser or any other direct or indirect wholly owned subsidiary of Gilead, for (x) $115.00 per Share (the Closing Amount), net to the seller in cash, without interest and subject to any required withholding of taxes, and (y) one contractual contingent value right (a CVR), which will represent the right to receive one contingent payment of $5.00 per CVR, in cash, without interest and subject to any required withholding of taxes, payable upon the achievement of a specified milestone in accordance with the terms and subject to the conditions of a contingent value rights agreement (the CVR Agreement), to be entered into with a rights agent selected by Gilead and reasonably acceptable to us (the Rights Agent) (the Closing Amount plus one (1) CVR together, the Offer Price).

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